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Bone Marrow Transplant Program

Bone Marrow Transplants (BMT) for pediatric patients is a treatment option for cancerous and non-cancerous disorders.  75 percent of the 30 to 45 pediatric BMTs that are performed at Johns Hopkins are done for bone marrow-related cancers and 25 percent are performed on patients with non-malignant disorders.  Johns Hopkins is fully approved by the Children’s Oncology Group, the National Marrow Donor Program, the Foundation for the Accreditation of Cellular Therapies, and is a Kaiser Permanente Center of Excellence for pediatric BMT on the East Coast.

Bone marrow transplantation is one of the best forms of immunotherapy that is available today.  It can be effective, especially when chemotherapy is not, but at the same time, carries some of the most risks of severe side effects of any treatment.  Side effects may occur as a result of the high doses of chemotherapy used to wipe out the patient’s immune system prior to transplanting the bone marrow.  They also occur as a result of radiation therapy and graft-versus-host disease.  Success of the transplant may depend on an effective preparative regimen that precedes the transplant and the degree of match.  The Johns Hopkins Pediatric BMT program is dedicated to optimizing the success of BMT while minimizing these risks.

Over the past 10 years, fatal complications after BMT have fallen by one-third due to:
• Matches:
Improvements in HLA typing – we can now identify transplant donors who match better

• Support: Supportive care has improved to help patients overcome side effects, for example, with more effective anti-fungal and antiviral medications

• GVH: The prevention and treatment of graft-versus-host disease have improved

 Tolerization: Johns Hopkins has developed a method to make donor bone marrow more tolerant to the patient, thus reducing the chance of serious graft-versus-host disease.  Typically, the chemotherapy drug cyclophosphamide is given as preparation before a transplant.  Our new approach is to use this drug at a critical time after the marrow graft is given to eliminate the cells that cause graft rejection and graft-versus-host disease. We are using this new method to decrease morbidity of transplant procedures and reduce the need for long-term immune suppression.

BMT for Cancer

  • Relapsed Acute Lymphocytic Leukemia (ALL): ALL is the most common pediatric malignancy, and is usually curable with chemotherapy alone; however, when children with ALL experience relapse, BMT often offers the best second chance for cure.
  • Acute Myelogenous Leukemia (AML) – BMT is often used as part of the initial therapy of patients with a sibling whose bone marrow is a match; in addition, BMT may be used as curative therapy for AML in the event initial therapy fails.
  • Chronic Myelogenous Leukemia – While the targeted therapy Gleevec is a primary treatment for adults with CML, the long-term effects of the drug are unknown for children.  BMT remains a curative treatment option for children with CML who have well-matched donors.
  • Recurrent or refractory Lymphoma – BMT is typically used for disease that has recurred or has not responded well to initial therapy.  The patient’s own marrow stem cells may often be used to cure this disease.
  • Neuroblastoma – A subset of these common childhood solid tumors are at high risk for relapse due to properties of the cells, disease staging, and age of the child, and for these patients, autologous BMT contributes to the chance of cure, as part of a multimodal approach including chemotherapy, surgery, radiation, differentiation therapy, and immunotherapy.
  • Desmoplastic Small Round Cell Tumors – a rare tumor that occurs most often in the abdomen of adolescents and young adults.
  • Ewing Sarcoma – a sarcoma that is sensitive to chemotherapy and radiation; some patients with metastatic or recurrent disease may benefit from BMT.
  • Alveolar Rhabdomyosarcoma – these cancers of skeletal muscle may occur in any part of the body, and the use of BMT is being explored for patients with high-risk disease in conjunction with treatments attacking the cancer stem cell.
  • Wilms Tumor – BMT may be used for selected patients with a high-risk pattern of relapse.
  • Brain Tumors – BMT is used in selected cases of aggressive, chemosensitive pediatric brain cancer.  In some cases, high-dose chemotherapy with marrow stem cell rescue may be a better alternative to radiation in children given the delicate nature of their young, growing brains.
  • Osteosarcoma – High-dose radioisotope therapy targeted to these bone tumors is being explored in combination with high-dose chemotherapy with marrow stem cells for patients with otherwise incurable disease.

BMT for Non-Malignant Diseases

  • Severe Aplastic Anemia (95 percent success rate in children with a matched sibling donor)
  • Fanconi Anemia and other inherited marrow failure syndromes
  • Sickle Cell Anemia
  • Thalassemia
  • Severe Combined Immune Deficiency, Wiskott-Aldrich Syndrome, and other congenital immune deficiencies
  • Granulocyte Disorders
  • X-Linked Autoimmunity-Allergic Dysregulation Syndrome (XLAAD), Autoimmune Enteropathy, Rasmussen’s Syndrome, and other severe autoimmune diseases
  • Adrenoleukodystrophy and other metabolic diseases

What to Expect
STEP 1: Pre-transplant Evaluation (1 week, outpatient)
STEP 2: Preparative Regimen (1 week, inpatient)
STEP 3: Engraftment (2 – 3 weeks, plus additional time as needed for recovery from side effects, inpatient)
STEP 4: Post-transplant Follow-up (2 months, outpatient) 

STEP 1: Pre-transplant Evaluation
• Diagnosis
• Disease Status
• Organ Function
• Donor Health and Safety

Evaluate Active/Latent Infections
Identify suitably matched donor or store patient stem cells

STEP 2: Preparative Regimen
BMT patients receive either chemotherapy alone or chemotherapy with radiation for one week as an inpatient at the Johns Hopkins Children’s Center.  Medications are given to prevent nausea and reduce the risk of infection and other side effects.

STEP 3: Engraftment
The procedure for transplanting bone marrow into a patient is similar to a blood transfusion.  It is given intravenously over a few hours during the patient’s hospital stay. 

Grafts may be:
1) allogeneic: from healthy donor, related or unrelated, or
2) autologous: from the patient’s own bone marrow or mobilized blood stem cells

After the transplanted marrow takes root and begins to manufacture new blood cells, it takes 2 -3 weeks for patients to begin producing enough blood cells to be managed as outpatients.  Johns Hopkins Children’s Center rooms in the Children’s Cancer Foundation Inpatient Unit are equipped with positive pressure HEPA filters that limit microbes to the same air quality as in operating rooms.  Once a patient leaves the hospital, it takes 6 to 12 months or more for the entire immune system to return to a normal state.  During that time, patients are more susceptible to infections, and precautions include wearing high-filtration masks, avoiding crowds and construction sites, and eating a low microbial diet.  Antibiotics are prescribed if fever develops.  Patients receive blood transfusions until their marrow produces sufficient red blood cells and platelets.  Management of nutrition, prevention and recovery from infection, prevention and treatment of graft-versus-host disease, and recovery of physical activity all begin in the hospital and continue in the outpatient setting.

STEP 4: Post-transplant Follow-up

Upon initial discharge from the hospital, patients will have 3 to 5 outpatient appointments every week at the Johns Hopkins Children’s Cancer Foundation Outpatient Unit.  During these appointments, patients may receive blood transfusions and other treatments.  Bone marrow testing is performed to assess the success of the transplant.  Cells in circulating blood are monitored for the presence of cancer and donor cells.

Typically, by about three months post-transplant, patients can return to the care of their referring physician, and they continue to be followed by Johns Hopkins pediatric oncologists at intervals until two years after BMT, and as needed.  Chronic graft-versus-host disease, which occurs when the donor’s immune system reacts against the patient, most often begins within the first two years after transplant.  Patients are monitored through young adulthood for late effects of cancer therapies, growth and development, bone density, and cardiac, renal and endocrine health.

If a patient’s disease returns after transplant, options may include donor lymphocyte infusions, a second transplant, chemotherapy, and palliative treatments.
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