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Pleuropulmonary Blastoma - Childhood - Introduction

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2016

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. To see other pages, use the menu. Think of that menu as a roadmap to this full 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 in this early form. It occurs in very young children, who on average are 10 months old. It has a better chance of being successfully treated than Types II and III, described below.

  • Type Ir is similar to Type I, but it does not have cancerous cells. 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 intensive chemotherapy.

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, including more information about genetic factors in Risk Factors.

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If you would like more of an introduction, explore this related item. Please note this link will take you to another section on Cancer.Net:

  • Cancer.Net Patient Education Video: View a short video led by an ASCO expert in childhood cancer that provides basic information and areas of research.

The next section in this guide is Statistics. It helps explain how many people are diagnosed with this disease and general survival rates. Or, use the menu to choose another section to continue reading this guide.   

Pleuropulmonary Blastoma - Childhood - Statistics

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2016

ON THIS PAGE: You will find information about how many children are diagnosed with this type of tumor each year. You will also learn some general information on surviving the disease. Remember, survival rates depend on several factors. To see other pages, use the menu.

PPB is rare. PPB occurs most often in children younger than 12, with most kids diagnosed between the ages of 3 to 4. PPB is rarely diagnosed in teenagers and adults. 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 of children with PPB is hard to estimate, given the rare nature of this disease. Talk with your doctor about your child’s prognosis and what individual factors it is based on.

It is important to remember that statistics on how many children survive PPB are an estimate. Doctors cannot say for sure how long any child will live with PPB. Estimates may not represent advances made in the treatment or diagnosis of PPB. Learn more about understanding statistics.

Source: International PPB Registry.

The next section in this guide is Risk Factors. It explains what factors may increase the chance of developing this disease. Or, use the menu on the side of your screen to choose another section to continue reading this guide.

Pleuropulmonary Blastoma - Childhood - Risk Factors

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2016

ON THIS PAGE: You will find out more about the factors that increase the chance of developing this type of tumor. To see other pages, use the menu.

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.

The cause of PPB is not known. Most often, it develops randomly. For most children with PPB, there may have been no personal or family history of the disease before the PPB occurred; in other words, there was no reason to suspect that the child would develop PPB. Most family members of children with PPB are healthy. The following factors may raise a person’s risk of developing PPB.

  • Cysts. Early stages of PPB appear as cysts. However, most children with lung cysts do not have PPB.

  • Family history/genetics. About 2 out of 3 of patients with  PPB have a genetic mutation of the DICER1 RNase-IIIb gene, called a germ line mutation. About 80% of patients inherited this gene 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 body changes or medical problems this disease can cause. Or, use the menu to choose another section to continue reading this guide.   

Pleuropulmonary Blastoma - Childhood - Symptoms and Signs

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2016

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

Children with PPB may experience the following symptoms or signs. Sometimes, children with PPB do not have any of these changes. Or, the cause of a symptom may be another medical condition that is a not tumor. Because PPB is so rare, doctors often believe at first 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. However, there are many other causes of pneumothorax.

  • Symptoms of PPB may be exactly the same as symptoms of pneumonia, a lung/respiratory infection. These symptoms may include generally feeling unwell, cough, 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 do not have an infection, but 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 you experience, please talk with your child’s doctor. Your 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 find 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. This may also be called symptom management, palliative care, or supportive care. Be sure to talk with the health care team about 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. Or, use the menu to choose another section to continue reading this guide.  

Pleuropulmonary Blastoma - Childhood - Diagnosis

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2016

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

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 whether 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.

This list describes options for diagnosing PPB, and not all tests listed will be used for every person.

The diagnosis of PPB depends on an examination under a microscope of material from inside the chest, either cyst material or solid tumor tissue. 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 an examination of a piece of the tumor 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 creates a 3-dimensional picture of the inside of the body using x-rays taken from different angles. A computer then combines these images into a detailed, cross-sectional view that shows any abnormalities or tumors. A CT scan can also 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 to swallow. After a chest x-ray shows something abnormal in the lungs, a CT scan is the best method for obtaining more information.

  • Magnetic resonance imaging (MRI). An MRI uses magnetic fields, not x-rays, to produce detailed images 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 to swallow. An MRI is not a very good way to look inside the chest, 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 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 gray to the camera, and areas of injury, such as those caused by a tumor, appear dark.

After diagnostic tests are done, your child’s doctor will review all of 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 extent of the disease. Or, use the menu to choose another section to continue reading this guide.  

Pleuropulmonary Blastoma - Childhood - Stages

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2016

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 to decide 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 year and is made up of mostly cysts, with almost no small, solid tumors that are 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, and occurs most commonly in children around 3 years old.

Type III: Type III PPB is a solid cancerous tumor, and 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.

Recurrent: Recurrent cancer is cancer has come back after treatment. If there is a recurrence, the tumor may need to be staged again using the criteria above in a process called restaging.

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 Treatment Options. Or, use the menu to choose another section to continue reading this guide.  

Pleuropulmonary Blastoma - Childhood - Treatment Options

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2016

ON THIS PAGE: You will learn about the different ways doctors use to treat children with PPB. To see other pages, use the menu.

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’s why more than 60% of children with cancer are treated as part of a clinical trial. Clinical trials are research studies that compare the standard of care with newer approaches to treatments that may be more effective. The “standard of care” is the best treatments known. Clinical trials may test such approaches as a new drug, a new combination of standard 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.

Treatment overview

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.

Descriptions of treatment options for PPB are listed 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 may also include treatment for symptoms and side effects, an important part of medical care. Surgery and chemotherapy, and sometimes radiation therapy, are used to treat PPB. If all of the tumor cannot be removed during surgery, chemotherapy and/or radiation therapy may be used to kill the remaining cells.

Take time to learn about all of your child’s treatment options and be sure to ask questions about things that are unclear. Also, talk about the goals of each treatment with your child’s doctor and what your child can expect while receiving the treatment. 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 performed before surgery to reduce the size of the tumor. This is called pre-operative chemotherapy.

Side effects from surgery for PPB can vary. Talk with your child’s doctor about possible short-term and long-term side effects. Learn more about the basics of cancer surgery.

Chemotherapy

Chemotherapy is the use of drugs to destroy tumor cells, usually by stopping the tumor cells’ ability to grow and divide. Chemotherapy is given by a medical oncologist, a doctor who specializes in treating cancer with medication.

Systemic chemotherapy is delivered through the bloodstream to reach cancer cells throughout the body. Common ways to give chemotherapy include an intravenous (IV) tube placed into a vein using a needle or in a pill or capsule that is swallowed.

A chemotherapy regimen, 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 combinations of different drugs 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 can be treated with chemotherapy, although surgery is more common. Chemotherapy is sometimes used to reduce the risk of recurrence. 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 to destroy any remaining PPB cells; this is called adjuvant or post-operative chemotherapy. Sometimes, pre-operative chemotherapy is given.

Common drugs for Type 1 PPB include: cyclophosphamide (Neosar), vincristine (Vincasar) and dactinomycin (Cosmegen). Specific drugs for Types II and III often includes 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 once treatment is finished.

Learn more about the basics of chemotherapy and preparing for treatment. The medications used to treat PPB are continually being evaluated. Talking with your child’s doctor is often the best way to learn about the medications prescribed for your child, their purpose, and their potential side effects or interactions with other medications. Learn more about your child’s prescriptions by using searchable drug databases.

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 usually consists of a specific number of treatments given over a set period of time.

Not every child with PPB receives radiation therapy. The decision to use radiation therapy is made if 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 radiation therapy.

Getting care for symptoms and side effects

PPB and its treatment often cause side effects. In addition to treatment to slow, stop, or eliminate the tumor, an important part of care is relieving a person’s symptoms and side effects. This approach is called palliative or supportive care, and it includes supporting the patient with his or her physical, emotional, and social needs.

Palliative care is any treatment that focuses on reducing symptoms, improving quality of life, and supporting patients and their families. Any person, regardless of age or type and stage of cancer, may receive palliative care. It works best when palliative care is started as early as needed in the cancer treatment process. People often receive treatment for the tumor and treatment to ease side effects at the same time. In fact, patients who receive both often have less severe symptoms, better quality of life, and report they are more satisfied with treatment.

Palliative treatments vary widely and often include medication, nutritional changes, relaxation techniques, emotional support, and other therapies. Your child may also receive palliative treatments similar to those meant to eliminate the tumor, such as chemotherapy, surgery, or radiation therapy. Talk with your child’s doctor about the goals of each treatment in the treatment plan.

Before treatment begins, talk with your child’s health care team about the possible side effects of the specific treatment plan and palliative care options. And during and after treatment, be sure to tell your child’s doctor or another health care team member if your child is experiencing a problem so it is addressed as quickly as possible. Learn more about palliative care.

Remission and the chance of recurrence

A remission is when a tumor 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 does return after the original treatment, it is called recurrent tumor. It may come back in the same place, meaning it is a local recurrence, or nearby, which is a regional recurrence. If it comes back in another place, it is a distant recurrence.

When this occurs, a 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.

When a tumor recurs, patients and their families often experience emotions such as disbelief or fear. Families are encouraged to talk with their health care team about these feelings and ask about support services to help them cope. Learn more about dealing with recurrence.

Advanced PPB

Advanced PPB is when the disease 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. PPB that has spread is rarely treated with surgery; other treatments will be recommended. 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.

If treatment fails

Although treatment is successful for the majority of children with cancer, sometimes it is not. If a child’s tumor cannot be cured or controlled, this is called advanced or terminal disease. This diagnosis is stressful, and advanced cancer may be difficult to discuss. However, it is important to have open and honest conversations with your child’s doctor and health care team to express your family’s feelings, preferences, and concerns. The health care team is there to help, and many team members have special skills, experience, and knowledge to support patients and their families.

Parents or guardians are encouraged to think about where the child would be most comfortable: at home, in a home-like setting elsewhere, in the hospital, or in a hospice environment. Hospice care is a type of palliative care for people who are expected to live less than 6 months. It is designed to provide the best possible quality of life for people who are near the end of life. Nursing care and special equipment can make staying at home a workable alternative for many families. Some children may be happier if they can arrange to 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. Or, use the menu to choose another section to continue reading this guide.  

Pleuropulmonary Blastoma - Childhood - About Clinical Trials

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2016

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. To see other pages, use the menu.

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. In fact, every drug that is now approved by the U.S. Food and Drug Administration (FDA) was tested in clinical trials.

Many clinical trials focus on new treatments. Researchers want to learn if a new treatment is safe, effective, and possibly better than the treatment doctors use now. 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 is no guarantee that the new treatment will be safe, effective, or better than what doctors use now.

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. There are also clinical trials studying ways to prevent cancer.

Deciding to join a clinical trial

Patients decide to participate in clinical trials for many reasons. For some patients, a clinical trial is the best treatment option available. Because standard treatments are not perfect, patients 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 the only way to make progress in treating PPB. Even if they do not benefit directly from the clinical trial, their participation may benefit future children with PPB.

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. During informed consent, the doctor should list all of the patient’s options, so that the person understands how the new treatment differs from the standard treatment. The doctor must also list all of the risks of the new treatment, which may or may not be different from the risks of standard treatment. Finally, the doctor must explain what will be required of each patient in order to participate in the clinical trial, including the number of doctor visits, tests, and the schedule of treatment.

Patients who participate in a clinical trial may stop participating at any time for any personal or medical reason. 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.

The next section in this guide is Latest Research. It explains areas of scientific research currently going on for this type of cancer. Or, use the menu to choose another section to continue reading this guide.    

Pleuropulmonary Blastoma - Childhood - Latest Research

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2016

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

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 diagnostic and treatment options best for your child.

  • Drug combinations. The experimental drug gefitinib (Iressa) is being tested in combination with irinotecan (Camptosar) and vincristine (Vincasar) to treat PPB in children. Cefixime (Suprax) and cefpodoxime (Vantin) are also added to the combination to reduce severe diarrhea for some patients.

  • Genetic causes of PPB. As explained in Risk Factors, researchers are continuing to investigate the link between a genetic mutation to DICER1 and familial PPB.

    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. This registry has approval from the participating institutions’ Institutional Review Boards to ensure the protection of patients’ privacy.

  • Treatment guidelines. Currently, there are no large-scale organized treatment schedules for PPB because it is so rare. Individual doctors use 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 recommendations for treating people with PPB.

  • Screening guidelines. “Screening” is used to look for cancer before signs or symptoms appear.

    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 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 mutations are also being evaluated for screening guidelines.

    As outlined in Risk Factors, DICER1 mutations are associated other cancers and tumors besides PPB.  Below is a list of several of those cancers, along with current screening options for people already diagnosed with PPB. However, it is important to note that this is an area of active research. Options include:

    • Cystic nephroma:

      • Baseline kidney CT scan or ultrasound examination in a patient diagnosed with PPB. However, The negative predictive value of a normal kidney CT or ultrasound examination for development of CN is not known.

      • In a patient of any age, especially those younger than 4 years, annual abdominal examination and monitoring for abdominal pain, swelling, or hematuria.

    • Thyroid gland neoplasia

      • Physical examination of the thyroid gland in a patient of any age

        • If the thryoid is not symmetrical and/or a nodule is detected, the patient may have a neck ultrasound examination. Results will indicate whether the doctor will monitor the patient or recommend a biopsy of the thyroid.

        • If no nodules are detected, continue annual physical examination. The doctor may also recommend repeating the thyroid ultrasound every 3 to 5 years.

      • Ultrasound examination of the neck may be recommended if the patient has previously received chemotherapy or as a baseline if the patient is going to have chemotherapy soon

      • Blood testing for thyroid functioning may be recommended, based on  clinical signs and the patient’s symptoms of an overactive or underactive thyroid gland.

    • Ovarian stromal tumors

      • Physical examination for girls/women of any age for signs and symptoms of early development of puberty or virilization (development of male puberty characteristics), and/or masses in the abdomen or pelvis. If any are found, the doctor will recommend appropriate imaging and laboratory evaluations.

      • Imaging may include abdominal-pelvic ultrasound examination, MRI, or CT scan

      • Laboratory testing may include serum markers AFP, beta-HCG, LDH, inhibin A and B, estradiol, testosterone, CA125, and serum electrolytes including calcium levels.

        Note: There is no current recommendation for laboratory screening in the absence of a mass or clinical findings of sex hormone excess.

      • For women and the parents of young girls with a DICER1 germline mutation, doctors will talk with you about the possible signs and symptoms of ovarian stromal tumors, such as abdominal distension, early puberty, amenorrhea (lack of menstruation), and signs of virilization.

    • Ciliary body medulloepithelioma (CBME)

      • Physical examination of young children for this type of cancer,  including measurement of visual acuity (how clear vision is)

      • Visual examination of the eyes and surrounding areas.

    • Botryoid embryonal rhabdomyosarcoma (ERMS).

      • In infants, children, and young adults when signs/symptoms of hematuria (blood in the urine) and/or abnormal vaginal bleeding are present, doctors may perform an endoscopic evaluation of the bladder and/or direct visualization of the cervix.

    • Nasal chondromesenchymal hamartoma (NCMH).

      • In patients of any age, the doctor will examine the body’s various systems including respiratory and feeding difficulties, rhinorrhea, epistaxis, visual disturbances, and otitis media.

      • The doctor may recommend a nasal endoscopy if there are ophthalmologic signs (such as ophthalmoplegia, proptosis, ptosis, hypotropia, and enophthalmos) resulting from orbital involvement of the tumor

    • Pituitary blastoma.

      • Brain MRI for patients with a DICER1 germline pathogenic variant when there are signs of too much of the hormone cortisol

    • Pineoblastoma.

      • Brain MRI for patients with a DICER1 germline pathogenic variant with signs of increased intracranial pressure such as headache, full fontanel (soft spot in skull), vomiting, and lethargy, or other neurologic defects including upgaze paralysis (problems with eye movement) and nystagmus (rapid eye movements that the person can’t control).

  • Palliative care. Clinical trials are underway to find better ways of reducing symptoms and side effects of current PPB treatments to improve patients’ comfort and quality of life.

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 in how to cope with the physical, emotional, and social changes that cancer and its treatment can bring. Or, use the menu to choose another section to continue reading this guide.  

Pleuropulmonary Blastoma - Childhood - Coping with Treatment

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2016

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. To see other pages, use the menu.

Every treatment can cause side effects or changes to your child’s body and how he or she feels. For many reasons, people don’t 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 health care team will work to prevent and relieve side effects. Doctors call this part of cancer treatment “palliative care.” It is an important part of your child’s treatment plan, regardless of his or her 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 Treatment Options section. Learn more about side effects of cancer and its treatment, along with ways to prevent or control them. Changes to your physical health depend on several factors, including the tumor’s stage, the length and dose of treatment, and your child’s general health.

Sometimes, physical 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 as well as physical effects after a cancer diagnosis. This may include dealing with difficult emotions, such as anxiety or anger, or managing your stress level. Sometimes, patients have problems expressing how they feel to their loved ones, or people don’t know what to say in return.

Patients and their families are encouraged to share their feelings with a member of their health care team. 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 financial effects

Treatment can be expensive. It is often 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. Learn more about managing financial considerations, in a separate part of this website.

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

Learn more about caregiving.

Talking with your 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 are likely to happen?

  • What can we do to prevent or relieve them?

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

Also, ask how much care your child may need at home and with daily tasks during and after treatment. This can help you make a caregiving plan.

The next section in this guide is Follow-up Care. It explains the importance of check-ups after your child finishes cancer treatment. Or, use the menu to choose another section to continue reading this guide.  

Pleuropulmonary Blastoma - Childhood - Follow-Up Care

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2016

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. To see other pages, use the menu.

Care for children diagnosed with PPB doesn’t end when active treatment has finished. Your child’s health care team will continue to check to make sure the cancer has not returned, 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.

Learn more about the importance of follow-up care.

Watching for recurrence

One goal of follow-up care is to check for a recurrence. 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 doctor will also 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 originally 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 rapidly grow, and this possibility should be discussed with your child’s doctor, including what signs and symptoms to watch for.

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 and include physical problems, such as heart and lung problems and second cancers, and emotional and cognitive (memory, thinking, and attention) problems, such as anxiety, depression, and learning difficulties.

Based on the type of treatment your child received, the doctor will recommend what examinations and tests are needed to check for late effects, such as 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 the child’s medical information. The doctor will help you create this. That way, as the child enters adulthood, he or she has a clear, written history of the diagnosis, the treatment given, 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 once treatment is completed.

Some children continue to see their oncologist, while others transition back to the general care of their family 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 your health care team about your child’s ongoing medical care and any concerns you have about his or her 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 him or her, as well as 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 his or her lifetime.

The next section in this guide is Survivorship. It describes how to cope with challenges in everyday life after a PPB diagnosis. Or, use the menu to choose another section to continue reading this guide.  

Pleuropulmonary Blastoma - Childhood - Survivorship

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2016

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

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 patient and his or her 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 of how to cope with everyday life.

One source of stress may occur when frequent visits to the health care team end following 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 as new worries and challenges surface over time, such as any late effects of treatment, educational issues, 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, and

  • 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 your health care team, individual counseling, or asking for assistance at the learning resource center of the center where you 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 in this article.

Healthy living after cancer

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

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.

In addition, it is important that your child has recommended medical check-ups and tests (see Follow-up Care) to take care of his or her health. 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 and productive as possible.

Talk with your 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 a lot of information and resources to help survivors cope, including specific sections for children, teens, and young adults. There is also a main section on survivorship for people of all ages.

  • ASCO Answers Cancer Survivorship Guide: Get this 44-page booklet that helps people transition into life after treatment. It includes blank treatment summary and survivorship care plan forms. The booklet is available as a PDF, so it is easy to print out.

  • 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 Doctor to help start conversations with your child’s health care team. Or, use the menu to choose another section to continue reading this guide.  

Pleuropulmonary Blastoma - Childhood - Questions to Ask the Doctor

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2016

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

Talking often with the doctor 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 an e-list and other interactive tools to manage your child’s care.

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?

  • 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 he or she be able to attend school and perform his or her usual activities?

  • Is there anything we can do before starting treatment to avoid 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?

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?

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

Questions to ask about having radiation therapy or chemotherapy

  • What type of treatment is recommended?

  • What is the goal of this treatment?

  • How long will it take to give this treatment?

  • What side effects can my child expect during treatment?

  • 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 he or she need them?

  • 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 some more resources on this website beyond this guide that may be helpful to you. Or, use the menu to choose another section to continue reading this guide.  

Pleuropulmonary Blastoma - Childhood - Additional Resources

This section has been reviewed and approved by the Cancer.Net Editorial Board, 03/2016

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 Guide to Pleuropulmonary Blastoma. To go back and review other pages, use the menu.

Cancer.Net includes many other sections about the medical and emotional aspects of cancer, both 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.

Beyond this guide, 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 select another section to continue reading this guide.