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Pleuropulmonary Blastoma - Childhood - Introduction

Approved by the Cancer.Net Editorial Board, 06/2022

ON THIS PAGE: You will find some basic information about this disease and the parts of the body it may affect. This is the first page of Cancer.Net’s Guide to Pleuropulmonary Blastoma. Use the menu to see other pages. Think of that menu as a roadmap for this entire guide.

Pleuropulmonary blastoma (PPB) is a rare type of childhood lung tumor that begins in the chest, either in the lung tissue (pulmonary) or in the tissue that covers the lungs and the inside of the pleura, also called the chest cavity.

A tumor begins when healthy cells change and grow out of control, forming a mass called a tumor. A tumor can be cancerous or benign. A cancerous tumor is malignant, meaning it can grow and spread to other parts of the body. A benign tumor means the tumor can grow but will not spread.

There are 4 types of PPB:

  • Type I is made up of air pockets called cysts with early evidence that it is cancerous. However, it is difficult to find and diagnose PPB in this early form. Type I PPB occurs in very young children, who on average are 10 months old. It has a better chance of being successfully treated, usually with surgery, than other types of PPB, such as Types II and III, described below.

  • Type Ir is similar to Type I, but it does not have cancerous cells and occurs in patients older than in Type I. The “r” stands for regressing.

  • Types II and III generally occur in children ages 3 to 4. Types II and III are cancerous tumors and require more intensive therapy, which includes chemotherapy and surgery.

Many patients with PPB have a mutation of the DICER1 RNase-IIIb gene. More details on the different types of PPB are explained in other sections of this guide, including more information about genetic factors in Risk Factors.

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The next section in this guide is Statistics. It helps explain the number of children who are diagnosed with pleuropulmonary blastoma and general survival rates. Use the menu to choose a different section to read in this guide.

Pleuropulmonary Blastoma - Childhood - Statistics

Approved by the Cancer.Net Editorial Board, 06/2022

ON THIS PAGE: You will find information about the number of children who are diagnosed with pleuropulmonary blastoma (PPB) each year. You will also read general information on surviving the disease. Remember, survival rates depend on several factors. Use the menu to see other pages.

PPB is rare. PPB occurs most often in children younger than 7. PPB is rarely diagnosed in teenagers and adults. Type Ir PPB (see Introduction) may be found in people of any age. PPB occurs about equally in boys and girls.

The overall survival rate tells you what percent of children live a certain amount of time after the cancer is found. Percent means how many out of 100. The overall survival rate for children with PPB is hard to estimate because the disease is rare. It is estimated that about 89% of children with Type I PPB are successfully treated, while about 50% to 70% of those with Type II or Type III are. Type I PPB can recur, or come back, as Type II or III PPB. Talk with your doctor about your child’s prognosis, or chance of recovery, and what individual factors it is based on.

It is important to remember that statistics on the survival rates for children with PPB are an estimate. The estimate comes from data based on the number of children with this cancer in the United States. Estimates may not reflect the results of advancements in how PBB in children is diagnosed or treated. Talk with your child’s doctor if you have any questions about this information. Learn more about understanding statistics.

Statistics adapted from the websites of the International Pleuropulmonary Blastoma/DICER1 Registry and the National Organization for Rare Disorders. (All sources accessed January 2022.)

The next section in this guide is Risk Factors. It describes the factors that may increase the chance of developing PPB. Use the menu to choose a different section to read in this guide.

Pleuropulmonary Blastoma - Childhood - Risk Factors

Approved by the Cancer.Net Editorial Board, 06/2022

ON THIS PAGE: You will find out more about the factors that increase the chance of developing pleuropulmonary blastoma (PPB). Use the menu to see other pages.

A risk factor is anything that increases a person’s chance of developing a tumor. Although risk factors often influence the development of a tumor, most do not directly cause a tumor. Some people with several risk factors never develop a tumor, while others with no known risk factors do.

About 70% of people with PPB have a genetic mutation of the DICER1 RNase-IIIb gene, which is a germline mutation. Germline mutations are inherited mutations that are passed down from one generation to the next in a family. About 80% of PPB patients inherited the DICER1 RNase-IIIb mutation from a parent.

The DICER1 syndrome is also associated with other cancers, such as cystic nephroma, cervical rhabdomyosarcoma, nasal chondromesenchymal hamartoma, ovarian stromal tumors, nodular thyroid hyperplasia, ciliary body medulloepithelioma, and pineoblastoma.

For screening information for PPB and other cancers related to DICER1, see the Latest Research section of this guide.

The next section in this guide is Symptoms and Signs. It explains what changes or medical problems PPB can cause. Use the menu to choose a different section to read in this guide.

Pleuropulmonary Blastoma - Childhood - Symptoms and Signs

Approved by the Cancer.Net Editorial Board, 06/2022

ON THIS PAGE: You will find out more about changes and other things that can signal a problem that may need medical care. Use the menu to see other pages.

Children with pleuropulmonary blastoma (PPB) may experience the following symptoms or signs. Symptoms are changes that you can feel in your body. Signs are changes in something measured, like by taking your blood pressure or doing a lab test. Together, symptoms and signs can help describe a medical problem. Sometimes, children with PPB do not have any of the symptoms and signs described below. Or, the cause of a symptom or sign may be a medical condition that is a not tumor. Because PPB is so rare, doctors often believe at first examination that the symptoms are caused by a common childhood illness.

There are 2 common sets of symptoms that may indicate PPB:

  • Sudden, stressful breathing may be caused by air escaping from the lung cysts into the chest cavity. This is called pneumothorax, meaning there is air in the chest cavity. It is important to note that there are many other causes of pneumothorax.

  • Symptoms of PPB may be exactly the same as symptoms of pneumonia, which is a lung/respiratory infection. These symptoms may include feeling generally unwell, a cough, a fever, and pain in the chest. Even when a chest x-ray is done, it may be first interpreted as pneumonia. It is common for children with PPB to have been treated for 2 to 3 weeks for pneumonia before more tests show that they likely have a tumor in the chest.

In addition, sometimes a lung cyst or tumor may be found when a chest x-ray is taken for another reason. See the Diagnosis section for more information on chest x-rays.

If you are concerned about any changes your child experiences, please talk with your child’s doctor. Your child's doctor will ask how long and how often your child has been experiencing the symptom(s), in addition to other questions. This is to help figure out the cause of the problem, called a diagnosis.

If PPB is diagnosed, relieving symptoms remains an important part of your child’s medical care and treatment. Managing symptoms may also be called "palliative care" or "supportive care." It is often started soon after diagnosis and continued throughout treatment. Be sure to talk with your child's health care team about the symptoms your child experiences, including any new symptoms or a change in symptoms.

The next section in this guide is Diagnosis. It explains what tests may be needed to learn more about the cause of the symptoms. Use the menu to choose a different section to read in this guide.

Pleuropulmonary Blastoma - Childhood - Diagnosis

Approved by the Cancer.Net Editorial Board, 06/2021

ON THIS PAGE: You will find a list of common tests, procedures, and scans that doctors use to find the cause of a medical problem. Use the menu to see other pages.

Doctors use many tests to find, or diagnose, a tumor. They also do tests to learn if the tumor has spread to another part of the body from where it started. If this happens, it is called metastasis. For example, imaging tests can show if the tumor has spread. Imaging tests show pictures of the inside of the body. Doctors may also do tests to learn which treatments could work best.

For most types of cancer, a biopsy is the only sure way for the doctor to know if an area of the body has cancer. In a biopsy, the doctor takes a small sample of tissue for testing in a laboratory. If a biopsy is not possible, the doctor may suggest other tests that will help make a diagnosis.

How PPB is diagnosed

There are many tests used for diagnosing PPB. Not all tests described here will be used for every person. Your child’s doctor may consider these factors when choosing a diagnostic test:

  • The type of cancer suspected

  • Your child’s signs and symptoms

  • Your child’s age and general health

  • The results of earlier medical tests

The diagnosis of PPB depends on an examination under a microscope of material (either cyst material or solid tumor tissue) from inside the chest. In Type I, or cystic, PPB, the cysts appear only slightly abnormal, but very close evaluation shows that the walls of the cysts contain very small collections of cancerous cells. In Types II and III PPB, it is obvious when looking under a microscope that tissue inside the chest is cancerous, but because PPB is so rare, it may be difficult for the doctors to determine exactly what type of tumor it is. In Types II and III PPB, it can spread to the heart, so doctors might also check the great vessels of the heart and the chest cavity as well. Often, doctors will send tissue samples to other experts for help determining the correct diagnosis.

In addition to a physical examination, the following tests may be used to diagnose PPB:

  • Biopsy. A biopsy is the removal of a small amount of tissue for examination under a microscope. Other tests can suggest that a tumor is present, but only a biopsy can make a definite diagnosis. A pathologist then analyzes the sample(s). A pathologist is a doctor who specializes in interpreting laboratory tests and evaluates cells, tissues, and organs to diagnose disease.

  • X-ray. An x-ray is a way to create a picture of the structures inside of the body using a small amount of radiation.

  • Computed tomography (CT or CAT) scan. A CT scan takes pictures of the inside of the body using x-rays taken from different angles. A computer combines these pictures into a detailed, 3-dimensional, or 3-D, image that shows any abnormalities or tumors. A CT scan can be used to measure the tumor’s size. Sometimes, a special dye called a contrast medium is given before the scan to provide better detail on the image. This dye can be injected into a patient’s vein or given as a pill or liquid to swallow. After a chest x-ray shows something abnormal in the lungs, a CT scan is the best method for obtaining more information about PPB.

  • Magnetic resonance imaging (MRI). An MRI uses magnetic fields, not x-rays, to produce detailed pictures of the body. MRI can also be used to measure the tumor’s size. A special dye called a contrast medium is given before the scan to create a clearer picture. This dye can be injected into a patient’s vein or given as a pill or liquid to swallow. An MRI is not as good as a CT scan to look inside the chest for PPB, but it is often the best test for looking at other parts of the body to determine if the tumor has spread.

  • Bone scan. A bone scan uses a radioactive tracer to look at the inside of the bones. The amount of radiation in the tracer is too low to be harmful. The tracer is injected into a patient’s vein. It collects in areas of the bone and is detected by a special camera. Healthy bone appears lighter to the camera, and areas of injury, such as those caused by a tumor, stand out on the image.

After diagnostic tests are done, your child’s doctor will review the results with you. If the diagnosis is PPB, these results also help the doctor describe the tumor. This is called staging.

The next section in this guide is Stages. It explains the system doctors use to describe the disease. Use the menu to choose a different section to read in this guide.

Pleuropulmonary Blastoma - Childhood - Stages

Approved by the Cancer.Net Editorial Board, 06/2021

ON THIS PAGE: You will learn about how doctors describe a tumor’s growth or spread. This is called the stage. To see other pages, use the menu.

Staging is a way of describing where a tumor is located, if or where it has spread, and whether it is affecting other parts of the body. Doctors use diagnostic tests to find out the tumor's stage, so staging may not be complete until all of the tests are finished. Knowing the stage helps the doctor recommend what kind of treatment is best and can help predict a patient's prognosis, which is the chance of recovery. There are different stage descriptions for different types of tumors.

The following criteria help doctors describe PPB:

Type I: Type I PPB generally occurs in children younger than 1 and is made up of mostly cysts with almost no small, solid tumors, called nodules. A thin layer of the wall of the cyst may appear cancerous. Type I PPB usually has the best prognosis of the different types.

Type Ir: Type Ir is made up of cysts that do not contain cancerous cells.

Type II: Type II PPB contains both cysts and cancerous nodules. It occurs most commonly in children around 3 years old.

Type III: Type III PPB is a solid cancerous tumor. It occurs most often in children around 4 years old.

For Types II and III, a bone scan and MRI (see Diagnosis) may be necessary to detect the stage.

Advanced: Advanced PPB is when the tumor has spread beyond where it started. Often this means the tumor has spread within the chest cavity or to the diaphragm, the thin muscle under the lungs and heart that separates the chest from the abdomen. PPB can also spread through the bloodstream to other organs, including the bones, liver, and brain.

Recurrent: Recurrent cancer is cancer that has come back after treatment. If the cancer does return, there will be another round of tests to learn about the extent of the recurrence. These tests and scans are often similar to those done at the time of the original diagnosis.

Sources: Dehner LP: Pleuropulmonary blastoma is THE pulmonary blastoma of childhood, Seminars in Diagnostic Pathology, 11:144-51, 1994, and The International PPB Registry.

Information about the tumor’s stage will help the doctor recommend a specific treatment plan. The next section in this guide is Types of Treatment. Use the menu to choose a different section to read in this guide.

Pleuropulmonary Blastoma - Childhood - Types of Treatment

Approved by the Cancer.Net Editorial Board, 06/2021

ON THIS PAGE: You will learn about the different types of treatments doctors use for children with PPB. Use the menu to see other pages.

In general, tumors in children are uncommon. This means it can be hard for doctors to plan treatments unless they know what has been most effective in other children. That is why more than 60% of children with cancer are treated as part of a clinical trial. A clinical trial is a research study that tests a new approach to treatment. The “standard of care” is the best treatments known based on research studies. Clinical trials may test such approaches as a new drug, a new combination of existing treatments, or new doses of current therapies. The health and safety of all children participating in clinical trials are closely monitored.

To take advantage of these newer treatments, children with PPB should be treated at a specialized treatment center. Doctors at these centers have extensive experience in treating children and have access to the latest research. A doctor who specializes in treating children with cancer is called a pediatric oncologist. If a pediatric cancer center is not nearby, general cancer centers sometimes have pediatric specialists who are able to be part of your child’s care.

How PPB is treated

In many cases, a team of doctors works with a child and the family to provide care. This is called a multidisciplinary team. Pediatric cancer centers often have extra support services for children and their families, such as child life specialists, dietitians, physical and occupational therapists, social workers, and counselors. Special activities and programs to help your child and family cope may also be available.

The common types of treatments used for PPB are described below. Treatment options and recommendations depend on several factors, including the type and stage of the tumor, possible side effects, and the patient’s preferences and overall health. Your child’s care plan also includes treatment for symptoms and side effects, an important part of cancer care.

Surgery and chemotherapy, and sometimes radiation therapy, are the main treatment options for PPB. If all of the tumor cannot be removed during surgery, chemotherapy and/or radiation therapy may be used as well.

Take time to learn about all of your child’s treatment options and be sure to ask questions about things that are unclear. Talk with your child’s doctor about the goals of each treatment and what your child can expect while receiving the treatment. These types of talks are called "shared decision-making." Shared decision-marking is when you and your child's doctors work together to choose treatments that fit the goals of your child's care. Shared decision-making is particularly important for PPB because there are different treatment options. Learn more about making treatment decisions.

Surgery

Surgery is the removal of the tumor and some surrounding healthy tissue during an operation. It is the main treatment for PPB. A surgical oncologist is a doctor who specializes in treating a tumor using surgery.

If the tumor is too large to be completely removed, or if it is not possible to completely remove the tumor, chemotherapy may be given before surgery to reduce the size of the tumor. This is called pre-operative or neoadjuvant chemotherapy. Chemotherapy and sometimes radiation therapy (see below) may also be given after surgery to destroy any remaining cells, called adjuvant therapy.

Before surgery, talk with your health care team about the possible side effects from the specific surgery that your child may experience and what recovery will be like. Learn more about the basics of cancer surgery.

Chemotherapy

Chemotherapy is the use of drugs to destroy tumor cells, usually by keeping the cancer cells from growing, dividing, and making more cells.

A chemotherapy regimen, or schedule, which is your child’s treatment schedule, usually consists of a specific number of cycles given over a set period of time. A patient may receive 1 drug at a time or a combination of different drugs given at the same time. Chemotherapy is often used to treat childhood tumors, and even children as young as newborn infants can be safely treated for life-threatening tumors with chemotherapy.

In some patients, Type I PPB is treated with surgery, and the role of chemotherapy after surgery is debated. Talk with your child’s doctor about whether it is recommended in your child’s case. Chemotherapy is always recommended for Types II and III PPB. The doctor may perform surgery first and then give chemotherapy after the surgery to destroy any remaining PPB cells. This is called adjuvant or post-operative chemotherapy. Sometimes, pre-operative chemotherapy is given.

Common drugs for Types II and III PPB include: cyclophosphamide (Neosar), vincristine (Vincasar) and dactinomycin (Cosmegen). Common treatment regimens for Types II and III include actinomycin D (IVADo), doxorubicin (Adriamycin), ifosfamide (Ifex), and vincristine (Vincasar).

The side effects of chemotherapy depend on the individual and the dose used, but they can include fatigue, risk of infection, nausea and vomiting, hair loss, loss of appetite, and diarrhea. These side effects usually go away after treatment is finished.

Learn more about the basics of chemotherapy.

Radiation therapy

Radiation therapy is the use of high-energy x-rays or other particles to destroy tumor cells. A doctor who specializes in giving radiation therapy to treat a tumor is called a radiation oncologist.

The most common type of radiation treatment is called external-beam radiation therapy, which is radiation given from a machine outside the body. When radiation treatment is given using implants, it is called internal radiation therapy or brachytherapy. A radiation therapy regimen, or schedule, usually consists of a specific number of treatments given over a set period of time.

Not every child with PPB receives radiation therapy. Radiation therapy may be recommended when the child’s health care team believes that there are still cancerous cells remaining after surgery and chemotherapy.

Side effects from radiation therapy may include fatigue, mild skin reactions, upset stomach, and loose bowel movements. Most side effects go away soon after treatment is finished. However, radiation therapy can also interfere with normal bone growth and is associated with development of secondary cancers. Talk with your child’s doctor about the possible short-term and long-term side effects before treatment begins. Learn more about the basics of radiation therapy.

Physical, emotional, and social effects of cancer

Cancer and its treatment cause physical symptoms and side effects, as well as emotional, social, and financial effects. Managing all of these effects is called palliative care or supportive care. It is an important part of your child’s care that is included along with treatments intended to slow, stop, or eliminate the cancer.

Palliative care focuses on improving how your child feels during treatment by managing symptoms and supporting patients and their families with other, non-medical needs. Any person, regardless of age or type and stage of cancer, may receive this type of care. And it often works best when it is started right after a cancer diagnosis. People who receive palliative care along with treatment for the cancer often have less severe symptoms, better quality of life, and report that they are more satisfied with treatment.

Palliative treatments vary widely and often include medication, nutritional changes, relaxation techniques, emotional and spiritual support, and other therapies. Your child may also receive palliative treatments similar to those meant to get rid of the cancer, such as chemotherapy, surgery, or radiation therapy.

Before treatment begins, talk with your child’s doctor about the goals of each treatment in the treatment plan being recommended. You should also talk about the possible side effects of the specific treatment plan and palliative care options. Many patients also benefit from talking with a social worker and participating in support groups. Ask your doctor about these resources, too.

During treatment, your child’s health care team may ask you to answer questions about your child’s symptoms and side effects and to describe each problem. Be sure to tell the health care team if your child is experiencing a problem. This helps the health care team treat any symptoms and side effects as quickly as possible. It can also help prevent more serious problems in the future.

Learn more about the importance of tracking side effects in another part of this guide. Learn more about palliative care in a separate section of this website.

Advanced PPB

Advanced PPB is when the tumor has spread beyond where it started, mostly within the chest cavity or to the diaphragm, the thin muscle under the lungs and heart that separates the chest from the abdomen. PPB can also spread through the bloodstream to other organs, especially the bones, liver, and brain.

Areas where PPB has spread are found by imaging tests, such as a CT scan, MRI scan, or bone scan. See the Diagnosis section for more information on these tests. If 1 area where PPB has spread is found on a scan, it is likely that the PPB has spread to other areas that cannot be seen on the scan. PPB that has spread is only rarely treated with surgery; other treatments will be recommended. Palliative care will also be important to help relieve symptoms and side effects.

A diagnosis of advanced PPB is stressful and difficult. Members of your family are encouraged to talk how your feel with your child's health care team. It may be also helpful to talk with other caregivers, such as a through a support group or peer support program.

Remission and the chance of recurrence

A remission is when PPB cannot be detected in the body and there are no symptoms. This may also be called having “no evidence of disease” or NED.

A remission may be temporary or permanent. This uncertainty causes many people to worry that the cancer will come back. While many remissions are permanent, it is important to talk with your child’s doctor about the possibility of the tumor returning. Understanding your child’s risk of recurrence and the treatment options may help you feel more prepared if the tumor does return. Learn more about coping with the fear of recurrence.

If the tumor returns after the original treatment, it is called a recurrent tumor. It may come back in the same place (called a local recurrence), nearby (regional recurrence), or in another place (distant recurrence).

When this occurs, a new cycle of testing will begin again to learn as much as possible about the recurrence. After testing is done, your child’s doctor will talk with you about treatment options. Often the treatment plan will include the treatments described above, such as surgery, chemotherapy, and radiation therapy, but they may be used in a different combination or given at a different pace. Your child’s doctor may also suggest clinical trials that are studying new ways to treat this type of recurrent tumor. Whichever treatment plan you choose, palliative care will be important for relieving symptoms and side effects.

Additional treatment depends on where and when the tumor recurred and how it was initially treated. Surgery may be used to remove a new tumor. Chemotherapy and/or radiation therapy may also be recommended. Bone marrow/stem cell transplant may also be a treatment option for some children.

People with recurrent cancer sometimes experience emotions such as disbelief or fear. You are encouraged to talk with the health care team about these feelings and ask about support services to help you cope. Learn more about dealing with cancer recurrence.

If treatment does not work

Although treatment is successful for many children with cancer, sometimes it is not. Sometimes, PPB cannot be cured or controlled. This diagnosis is stressful and may be difficult to discuss. However, it is important to have open and honest conversations with your child’s health care team to express your family’s feelings, preferences, and concerns. The health care team has special skills, experience, and knowledge to support patients and their families and is there to help.

Hospice care is designed to provide the best possible quality of life for people who are are expected to live less than 6 months. Parents and guardians are encouraged to talk with the health care team about hospice options, which include hospice care at home, a special hospice center, or other health care locations. Nursing care and special equipment can make staying at home a workable option for many families. Some children may be happier and more comfortable if they can attend school part-time or keep up other activities and social connections. The child’s health care team can help parents or guardians decide on an appropriate level of activity. Making sure a child is physically comfortable and free from pain is extremely important as part of end-of-life care. Learn more about caring for a terminally ill child and advanced cancer care planning.

The death of a child is an enormous tragedy, and families may need support to help them cope with the loss. Pediatric cancer centers often have professional staff and support groups to help with the process of grieving. Learn more on grieving the loss of a child.

The next section in this guide is About Clinical Trials. It offers more information about research studies that are focused on finding better ways to care for people with PPB. Use the menu to choose a different section to read in this guide.

Pleuropulmonary Blastoma - Childhood - About Clinical Trials

Approved by the Cancer.Net Editorial Board, 06/2021

ON THIS PAGE: You will learn more about clinical trials, which are the main way that new medical approaches are tested to see how well they work. Use the menu to see other pages.

What are clinical trials?

Doctors and scientists are always looking for better ways to care for children with PPB. To make scientific advances, doctors create research studies involving volunteers, called clinical trials. Every drug that is now approved by the U.S. Food and Drug Administration (FDA) was tested in clinical trials.

Clinical trials are used for all types and stages of PPB. Many focus on new treatments to learn if a new treatment is safe, effective, and possibly better than the existing treatments. These types of studies evaluate new drugs, different combinations of existing treatments, new approaches to radiation therapy or surgery, and new methods of treatment.

Children who participate in clinical trials can be some of the first to get a treatment before it is available to the public. However, there are some risks with a clinical trial, including possible side effects and the chance that the new treatment may not work. People are encouraged to talk with their health care team about the pros and cons of joining a specific study.

Some clinical trials study new ways to relieve symptoms and side effects during treatment. Others study ways to manage the late effects that may happen a long time after treatment. Talk with your doctor about clinical trials for symptoms and side effects.

Deciding to join a clinical trial

People decide to participate in clinical trials for many reasons. For some people, a clinical trial is the best treatment option available. Because standard treatments are not perfect, people are often willing to face the added uncertainty of a clinical trial in the hope of a better result. Other patients volunteer for clinical trials because they know that these studies are a way to contribute to the progress in treating PPB. Even if they do not benefit directly from the clinical trial, their participation may benefit future children with PPB.

Insurance coverage and the costs of clinical trials differ by location and by study. In some programs, some of the patient’s expenses from participating in the clinical trial are reimbursed. In others, they are not. It is important to talk with the research team and your insurance company first to learn if and how your child's treatment in a clinical trial will be covered. Learn more about health insurance coverage of clinical trials.

Sometimes people have concerns that, in a clinical trial, their child may receive no treatment by being given a placebo or a “sugar pill.” The use of placebos in cancer clinical trials in this way is rare overall and not done at all in childhood cancer research. Find out more about placebos in cancer clinical trials.

Patient safety and informed consent

To join a clinical trial, parents and children must participate in a process known as informed consent. Informed consent means that parents give permission for their child to participate in a clinical trial and that teenagers give their consent to participate. During informed consent, the doctor should:

  • Describe all of the treatment options so that the person understands how the new treatment differs from the standard treatment.

  • List all of the risks of the new treatment, which may or may not be different from the risks of standard treatment.

  • Explain what will be required of each person in order to participate in the clinical trial, including the number of doctor visits, tests, and the schedule of treatment.

  • Describe the purposes of the clinical trial and what researchers are trying to learn.

Clinical trials also have certain rules called “eligibility criteria” that help structure the research and keep patients safe. You and the research team will carefully review these criteria together.

People who participate in a clinical trial may stop participating at any time for personal or medical reasons. This may include that the new treatment is not working or there are serious side effects. Clinical trials are also closely monitored by experts who watch for any problems with each study. It is important that parents and children participating in a clinical trial talk with their doctor and researchers about who will be providing their treatment and care during the clinical trial, after the clinical trial ends, and/or if the patient chooses to leave the clinical trial before it ends.

Finding a clinical trial

Research through clinical trials is ongoing for all types of cancer. For specific topics being studied for PPB, learn more in the Latest Research section.

Cancer.Net offers a lot of information about clinical trials in other areas of the website, including a complete section on clinical trials and places to search for clinical trials for a specific type of cancer.

PRE-ACT, Preparatory Education About Clinical TrialsIn addition, this website offers free access to a video-based educational program about cancer clinical trials, located outside of this guide.

The next section in this guide is Latest Research. It explains areas of scientific research for PPB. Use the menu to choose a different section to read in this guide.

Pleuropulmonary Blastoma - Childhood - Latest Research

Approved by the Cancer.Net Editorial Board, 06/2021

ON THIS PAGE: You will read about the scientific research being done to learn more about this type of tumor and how to treat it. Use the menu to see other pages.

Doctors are working to learn more about PPB, ways to prevent it, how to best treat it, and how to provide the best care to people diagnosed with this disease. The following areas of research may include new options for patients through clinical trials. Always talk with your child’s doctor about the best diagnostic and treatment options for your child.

  • Genetic causes of PPB. As explained in Risk Factors, researchers are continuing to investigate the link between a genetic mutation to DICER1 and PPB in families. Research into causes and treatment for a rare tumor like PPB requires collecting information from many hospitals. The International PPB Registry is the largest such collection of information on PPB in the world. (Please note this link takes you to a separate website.) This registry has approval from the participating institutions’ Institutional Review Boards to ensure the protection of patients’ privacy.

  • Treatment guidelines. Currently, there are no widely used treatment schedules for PPB because it is so rare. Individual doctors use research and their experience in treating similar conditions, such as soft-tissue sarcomas, to guide their PPB treatment recommendations. Plans are underway to create an international consortium of pediatric oncology specialists from around the world to consider treatment options and make specific recommendations for treating people with PPB.

  • Screening guidelines. Doctors consider screening for genetic testing for DICER1 in a person who has at least 1 major feature and 2 minor features as listed below:

    Major features
    :

           A diagnosis of PPB (any type)

           A lung cyst in childhood

           Thoracic embryonal rhabdomyosarcoma, a type of soft-tissue sarcoma

           Cystic nephroma, a benign kidney tumor

           Sarcoma in the genitourinary tract, including undifferentiated sarcoma

           Ovarian Sertoli-Leydig cell tumor

           Gynandroblastoma, a rare ovarian tumor

           Uterine, cervical, or ovarian embryonal rhabdomyosarcoma

           Neuroendocrine tumor in the genitourinary or gynecologic system

           Thyroid nodules or cancer in 2 or more first-degree relatives (such as a parent or child)

           Thyroid nodules or differentiated thyroid cancer in childhood

           Ciliary body medulloepithelioma, a type of childhood eye tumor

           Nasal chondromesenchymal hamartoma, a benign tumor in the nasal sinus area

           Pineoblastoma, a cancerous tumor of the brain’s pineal gland

           Pituitary blastoma, an aggressive tumor of the pituitary gland

           Minor features
:

           A lung cyst in an adult

           Renal (kidney) cysts

           Wilms tumor, a childhood kidney cancer

           Thyroid nodules or differentiated thyroid cancer in an adult

           Embryonal rhabdomyosarcoma not listed under "Major features" (see above)

           Neuroendocrine tumor that is poorly differentiated

           Undifferentiated sarcoma somewhere besides the genitourinary tract

           Macrocephaly, an overly large head size in childhood

           Childhood cancers associated with any Minor features

Source:
Clinical Cancer Research 2018 May 15;24(10):2251-2261. doi: 10 (Table 1).

If a person is found to have DICER1, doctors will consider screening them further for specific conditions based on specific signs and symptoms that are reported to the doctor or found on a physical exam to look for problems (including those listed above in Major and Minor Features) in these main body systems:

Lung system – Symptoms include abnormally rapid breathing, cough, fever, pain, and a collapsed lung. Screening tests include regular chest x-rays or a chest CT scan.

Thyroid gland – Symptoms include a nodule or growth on the thyroid gland that is visible or can be easily felt, persistent enlargement of neck lymph nodes, hoarseness, difficulty swallowing, neck pain, and cough. Screening tests include regular thyroid ultrasounds and regular physical exams to check the thyroid.

Gynecologic system – Symptoms include dark, coarse, male-pattern hair growth in women, development of other male characteristics such as a deepening voice in women, and abdominal pain, expansion, or a mass. Screening tests include regular pelvic and abdominal ultrasounds.

Kidneys/genitourinary system – Symptoms include an abdominal mass or pain, or blood in the urine. Screening tests include regular abdominal ultrasounds.

Gastrointestinal system – Symptoms include any sign of intestinal obstruction, such as cramps, constipation, vomiting, or abdominal swelling. Doctors will talk with a person about what screening tests may be appropriate for them.

Central nervous system (CNS) and the head and neck system – This includes symptoms that do not involve the thyroid gland (see above), such as headaches, vomiting, double-vision, inability to look upward, difficulty walking, premature puberty, Cushing’s syndrome, vision problems, and nasal obstruction. Screening tests include regular physical exams, annual ophthalmologic exam including visual acuity screening, as well as MRI for emergency symptoms inside the skull.

Source: Clinical Cancer Research 2018 May 15;24(10):2251-2261. doi: 10 (Table 2).

Doctors are looking at ways to screen children for PPB and other cancers related to DICER1 genetic mutations. For PPB, screening guidelines will help doctors know when it’s best to use a CT scan to look for possible lung cysts or tumors, particularly for children under the age of 3. MRIs of the brain for children with the DICER1 germline mutation are also being evaluated for screening guidelines.

Looking for More about Latest Research?

If you would like additional information about the latest areas of research regarding PPB, explore these related items that take you outside of this guide:

The next section in this guide is Coping with Treatment. It offers some guidance on how to cope with the physical, emotional, social, and financial changes that cancer and its treatment can bring. Use the menu to choose a different section to read in this guide.

Pleuropulmonary Blastoma - Childhood - Coping with Treatment

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ON THIS PAGE: You will learn more about coping with the physical, emotional, social, and financial effects of childhood cancer and its treatment. This page includes several links outside of this guide to other sections of this website. Use the menu to see other pages.

Every treatment can cause side effects or changes to your child’s body and how they feel. For many reasons, people do not experience the same side effects even when given the same treatment for the same type of tumor. This can make it hard to predict how your child will feel during treatment.

As your family prepares to start cancer treatment, it is normal to fear treatment-related side effects. It may help to know that your child’s health care team will work to prevent and relieve side effects. This part of cancer treatment is called “palliative care” or “supportive care.” It is an important part of your child’s treatment plan, regardless of their age or the stage of disease.

Coping with physical side effects

Common physical side effects from each treatment option for PPB are described within the Types of Treatment section. Learn more about side effects of cancer and its treatment, along with ways to prevent or control them. Changes to your child’s physical health depend on several factors, including the tumor’s stage, the length and dose of treatment, and your child’s general health.

It is important to discuss any new side effects or changes in existing side effects with your child’s health care team. Providing this information helps them find ways to treat or manage the side effects so your child feels more comfortable and can potentially keep any side effects from worsening.

You may find it helpful to keep track of your child’s side effects so you are prepared to discuss any changes with the health care team. Learn more about why tracking side effects is helpful.

Sometimes, side effects can last after treatment ends. Doctors call these long-term side effects. They call side effects that occur months or years after treatment late effects. Treating long-term side effects and late effects is an important part of care for childhood cancer survivors. Learn more by reading the Follow-Up Care section of this guide or talking with your child’s doctor.

Coping with emotional and social effects

Your family can have emotional and social effects after a cancer diagnosis. This may include dealing with a variety of emotions, such as anxiety or anger, or managing stress. Sometimes, people find it difficult to express how they feel to their loved ones. Some have found that talking to an oncology social worker, counselor, or member of the clergy can help them develop more effective ways of coping and talking about cancer.

You can also find coping strategies for emotional and social effects in a separate section of this website. This section includes many resources for finding support and information to meet your family’s needs.

Coping with the costs of cancer care

Treatment can be expensive. It can be a big source of stress and anxiety for families dealing with a cancer diagnosis. In addition to treatment costs, many people find they have extra, unplanned expenses related to their child’s care. Families are encouraged to talk about financial concerns with a member of their health care team. Learn more about managing financial considerations in a separate part of this website.

Coping with barriers to care

Different groups of people experience different rates of new cancer cases and experience different outcomes from their cancer. These differences are called “cancer disparities.” Disparities are caused in part by real-world barriers to quality medical care and more often negatively affect racial and ethnic minorities, poor people, sexual and gender minorities (LGBTQ+), adolescent and young adult populations, older adults, and people who live in rural areas or other underserved communities.

If your child is having difficulty getting the care they need, talk with a member of the health care team or explore other resources that help support medically underserved people.

Caring for a child with cancer

Family members and friends often play an important role in taking care of a person with PPB. This is called being a caregiver. As a parent or guardian, you are the primary caregiver for your child. However, friends and family members can give your family valuable support, even if they live far away.

When your child has PPB, you may have an additional range of responsibilities. These may include giving medications or managing symptoms and side effects. However, it is important to seek help from others. Below are some of the responsibilities your family or friends could help with:

  • Providing short-term care for your child

  • Giving support and encouragement

  • Assisting with meals or household chores

  • Helping with insurance and billing issues

A caregiving plan can help caregivers stay organized and help identify opportunities to delegate tasks to others. Ask how much care your child may need at home and with daily tasks during and after treatment. Use this 1-page fact sheet that includes an action plan to help make caregiving a team effort. This free fact sheet is available as a PDF, so it is easy to print.

Learn more about caregiving or read the ASCO Answers Guide to Caring for a Loved One in English or Spanish.

Talking with your child’s health care team about side effects

Before starting treatment, talk with your child’s doctor about possible side effects. Ask:

  • Which side effects are most likely?

  • When are they likely to happen?

  • What can we do to prevent or relieve them?

Be sure to tell your child’s health care team about any side effects that happen during treatment and afterward, too. Tell them even if you do not think the side effects are serious. This discussion should include physical, emotional, social, and financial effects of cancer.

Looking for More on How to Track Side Effects?

Cancer.Net Mobile app symptom tracker

Cancer.Net offers several resources to help you keep track of symptoms and side effects. Please note that these links will take you to other sections of Cancer.Net:

  • Cancer.Net Mobile: The free Cancer.Net mobile app allows you to securely record the time and severity of your child's symptoms and side effects. 

  • ASCO Answers Managing Pain: Get this 32-page booklet about the importance of pain relief that includes a pain tracking sheet to record how pain affects your child. The free booklet is available as a PDF, so it is easy to print.

  • ASCO Answers Fact Sheets: Read 1-page fact sheets on anxiety and depression, constipation, diarrhea, and rash that provide a tracking sheet to record details about the side effect. These free fact sheets are available as a PDF, so they are easy to print, fill out, and give to your health care team.

The next section in this guide is Follow-Up Care. It explains the importance of checkups after your child finishes cancer treatment. Use the menu to choose a different section to read in this guide.

Pleuropulmonary Blastoma - Childhood - Follow-Up Care

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ON THIS PAGE: You will read about your child’s medical care after treatment for PPB is finished and why this follow-up care is important. Use the menu to see other pages.

Care for children diagnosed with PPB does not end when active treatment has finished. Your child’s health care team will continue to check that the cancer has not come back, manage any side effects, and monitor your child’s overall health. This is called follow-up care. All children treated for PPB should have life-long, follow-up care.

Your child’s follow-up care may include regular physical examinations, medical tests, or both. Doctors want to keep track of your child’s recovery in the months and years ahead. Recommendations for follow-up care according to the risk of possible late effects of treatment have been developed by the Children's Oncology Group and can be found at CureSearch.org, a separate website.

Cancer rehabilitation may be recommended, and this could mean any of a wide range of services such as physical therapy, family or individual counseling, nutritional planning, and/or educational assistance. The goal of rehabilitation is to help survivors and their families regain control over many aspects of their lives and remain as independent as possible. Learn more about cancer rehabilitation.

Learn more about the importance of follow-up care.

Watching for recurrence

One goal of follow-up care is to check for a recurrence, which means that the cancer has come back. A tumor recurs because small areas of tumor cells may remain undetected in the body. Over time, these cells may increase in number until they show up on test results or cause signs or symptoms.

During follow-up care, a doctor familiar with your child’s medical history can give you personalized information about the risk of recurrence. Your child’s doctor will ask specific questions about your child’s health. Some children may have blood tests or imaging tests as part of regular follow-up care, but testing recommendations depend on several factors, including the type and stage of PPB first diagnosed and the types of treatment given.

If a recurrence happens, it is more likely to occur in the first few years after the original treatment. Therefore, regular screening should continue for 2 to 3 years, or possibly up to 5 years, after diagnosis. Very rarely, PPB returns after these standard periods of follow-up. It is important to note that PPB can grow rapidly, and this possibility should be discussed with your child’s doctor, including what signs and symptoms to watch for.

The anticipation before having a follow-up test or waiting for test results may add stress to you or a family member. This is sometimes called “scanxiety.” Learn more about how to cope with this type of stress.

Managing long-term and late side effects of childhood cancer

Sometimes, side effects may linger beyond the active treatment period. These are called long-term side effects. In addition, other side effects called late effects may develop months or even years afterwards. Late effects can occur almost anywhere in the body. They include physical problems, such as heart and lung problems and second cancers, which is a new cancer that happens in someone who has had cancer before. They also include emotional problems, such as anxiety and depression, and problems with memory, thinking, attention, and learning.

Based on the type of treatment your child received, the doctor will recommend what examinations and tests are needed to check for late effects. Follow-up care should address your child’s quality of life, including any developmental or emotional concerns.

The Children's Oncology Group (COG) has studied the physical and psychological effects that childhood cancer survivors face. Based on these studies, COG has created recommendations for long-term follow-up care for childhood, adolescent, and young adult cancer survivors that can be found on a separate website: www.survivorshipguidelines.org.

Keeping a child’s personal health record

You are encouraged to organize and keep a personal record of your child’s medical information. The doctor will help you create this. That way, as the child enters adulthood, they have a clear, written history of the diagnosis, the treatments, and the doctor’s recommendations about the schedule for follow-up care. ASCO offers forms to help create a treatment summary to keep track of the treatment of PPB your child received and develop a survivorship care plan when treatment is completed.

Some children continue to see their oncologist, while others transition back to the care of their primary care doctor or another health care professional. This decision depends on several factors, including the type and stage of PPB, side effects, health insurance rules, and your family’s personal preferences. Talk with the health care team about your child’s ongoing medical care and any concerns you have about their future health.

If a doctor who was not directly involved in your child’s medical care will lead the follow-up care, be sure to share the treatment summary and survivorship care plan forms with them and with all future health care providers. Details about the specific treatment given for PPB are very valuable to the health care professionals who will care for your child throughout their lifetime.

The next section in this guide is Survivorship. It describes how to cope with challenges in everyday life after a PPB diagnosis. Use the menu to choose a different section to read in this guide.

Pleuropulmonary Blastoma - Childhood - Survivorship

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ON THIS PAGE: You will read about how to cope with challenges in everyday life after your child’s cancer diagnosis. Use the menu to see other pages.

What is survivorship?

The word “survivorship” means different things to different people, but it often describes living with, through, and beyond cancer. In some ways, survivorship is one of the most complex aspects of the cancer experience because it is different for every child and their family.

After active treatment ends, children and their families may experience a mixture of strong feelings, including joy, concern, relief, guilt, and fear. Some people say they appreciate life more after a cancer diagnosis. Other families stay very anxious about their child’s health and become uncertain about coping with everyday life.

One source of stress may occur when frequent visits to the health care team end after completing treatment. Often, relationships built with the health care team provide a sense of security during treatment, and children and their families miss this source of support. This may be especially true when new worries and challenges surface over time, such as any late effects of treatment, learning or school problems, emotional challenges, sexual development and fertility concerns, and/or financial issues.

Every family faces different concerns and challenges. With any challenge, a good first step is being able to recognize each fear and talk about it. Effective coping requires:

  • Understanding the challenge your family is facing

  • Thinking through solutions

  • Asking for and allowing the support of others

  • Feeling comfortable with the course of action your family chooses

It may be helpful to join an in-person support group or online community of childhood cancer survivors. Support groups also exist for parents of children diagnosed with cancer. This allows you to talk with people who have had similar first-hand experiences. Other options for finding support include talking with a friend or member of the health care team, individual counseling, or asking for assistance at the learning resource center of the place where your child received treatment.

Changing role of caregivers

Parents, other family members, and friends may also go through periods of transition. A caregiver plays a very important role in supporting a child diagnosed with PPB, providing physical, emotional, and practical care on a daily or as-needed basis. Many caregivers become focused on providing this support, especially if the treatment period lasts for many months or longer.

However, as treatment is completed, the caregiver's role often changes. Eventually, the need for caregiving related to a child’s diagnosis will become much less or come to an end as your child gets older. Family counselors at pediatric cancer centers can help with this transition. You can also learn more about adjusting to life after caregiving.

Healthy living after cancer

Survivorship often serves as a strong motivator to make lifestyle changes, often for the whole family.

Children who have had PPB can enhance the quality of their future by following established guidelines for good health into and through adulthood, including not smoking, maintaining a healthy weight, eating well, managing stress, and participating in regular physical activity. Talk with the doctor about developing a plan that is best for your child’s needs. Learn more about making healthy lifestyle choices.

It is important that your child has recommended medical checkups and tests (see Follow-up Care) to take care of their health. 

Talk with the doctor to develop a survivorship care plan that is best for your child’s needs.

Looking for More Survivorship Resources?

For more information about cancer survivorship, explore these related items. Please note these links will take you to other sections of Cancer.Net:

  • Survivorship Resources: Cancer.Net offers information and resources to help survivors cope, including specific sections for children and teens and young adults. There is also a main section on survivorship for people of all ages.

  • ASCO Answers Cancer Survivorship Guide: Get this 48-page booklet that helps people transition into life after treatment. It includes blank treatment summary and survivorship care plan forms. The free booklet is available as a PDF, so it is easy to print.

  • Cancer.Net Patient Education Video: View a short video led by an ASCO expert that provides information about childhood cancer survivorship.

The next section offers Questions to Ask the Health Care Team to help start conversations with your child’s health care team. Use the menu to choose a different section to read in this guide.

Pleuropulmonary Blastoma - Childhood - Questions to Ask the Health Care Team

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ON THIS PAGE: You will find some questions to ask your child’s doctor or other members of the health care team, to help you better understand your child’s diagnosis, treatment plan, and overall care. Use the menu to see other pages.

Talking often with the health care team is important to make informed decisions about your child’s health care. These suggested questions are a starting point to help you learn more about your child’s care and treatment. You are also encouraged to ask additional questions that are important to you. You may want to print this list and bring it to your child’s next appointment, or download Cancer.Net’s free mobile app for a digital list and other interactive tools to manage your child’s care. It may also be helpful to ask a family member or friend to come with you to appointments to help take notes.

Cancer.Net Mobile app question tracker

Questions to ask after getting a diagnosis

  • What type of PPB has been diagnosed? What does this mean?

  • Can you explain my child’s pathology report, or laboratory test results, to me?

  • Where is the PPB in my child's body?

  • What is your familiarity with PPB and its treatment?

  • Do you collaborate with the International PPB Registry?

Questions to ask about choosing a treatment and managing side effects

  • What are my child’s treatment options?

  • What is the goal of each treatment? Is it to eliminate the tumor, help my child feel better, or both?

  • What clinical trials are available for my child? Where are they located, and how do I find out more about them?

  • What treatment plan do you recommend? Why?

  • What is my child’s prognosis?

  • Should I seek a second opinion?

  • Does your practice include multidisciplinary care?

  • Who will be part of my child’s health care team, and what does each member do?

  • Who will be leading my child’s overall treatment?

  • Do you attend expert meetings to discuss complicated tumor cases? What kinds of specialists attend such meetings?

  • What are the possible side effects of each treatment, both in the short term and the long term?

  • How will this treatment affect my child’s daily life? Will they be able to attend school and perform their usual activities?

  • Is there anything we can do before starting treatment to avoid or reduce late effects?

  • Could this treatment affect my child’s ability to become pregnant or have children in the future? If so, should I talk with a fertility specialist before cancer treatment begins?

  • If I am worried about managing the costs of medical care, who can help?

  • What support services are available to my child? To my family?

  • If I have questions or problems, who should I call?

Questions to ask about having surgery

  • What type of surgery will my child have? Will lymph nodes be removed?

  • How long will the operation take?

  • How long will my child be in the hospital?

  • Can you describe what my recovery from surgery will be like?

  • Who should I contact about any side effects my child experiences? And how soon?

  • What are the possible long-term effects of having this surgery?

Questions to ask about having chemotherapy or radiation therapy

  • What type of treatment is recommended?

  • What is the goal of this treatment?

  • How long will it take to give this treatment?

  • Will my child receive this treatment at a hospital or clinic?

  • What side effects can my child expect during treatment?

  • Who should I contact about any side effects my child experiences? And how soon?

  • What are the possible long-term effects of having this treatment?

  • What can be done to relieve the side effects?

Questions to ask about planning follow-up care

  • What is the chance that the tumor will come back? Should I watch for specific signs or symptoms?

  • What long-term side effects or late effects are possible based on the treatment for PPB that my child received?

  • What follow-up tests will my child need, and how often will those tests be needed?

  • How do I get a treatment summary and survivorship care plan to keep in my child’s personal records?

  • Who will be leading my child’s follow-up care?

  • What survivorship support services are available to my child? To my family?

The next section in this guide is Additional Resources. It offers more resources on this website that may be helpful to you. Use the menu to choose a different section to read in this guide.

Pleuropulmonary Blastoma - Childhood - Additional Resources

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ON THIS PAGE: You will find some helpful links to other areas of Cancer.Net that provide information about cancer care and treatment. This is the final page of Cancer.Net’s Guide to Pleuropulmonary Blastoma. Use the menu to go back and see other pages.

Cancer.Net includes many other sections about the medical and emotional aspects of a tumor for the person diagnosed and their family members and friends. This website is meant to be a resource for you and your loved ones from the time of diagnosis, through treatment, and beyond.

Here are a few links to help you explore other parts of Cancer.Net:

This is the end of Cancer.Net’s Guide to Pleuropulmonary Blastoma. Use the menu to choose a different section to read in this guide.