Stem Cell Transplantation Program (Cellular Therapy)

The Hematopoietic Stem Cell Transplant (HSCT) Program has been an integral part of the Division of Hematology/Oncology and Stem Cell Transplantation since 1991 and has made possible hundreds of successful stem cell transplants for a wide variety of malignant and nonmalignant disorders. The program is fully accredited by the Foundation for Accreditation of Cellular Therapy (FACT) and is staffed by two full-time attending physicians, two nurse practitioners, a social worker and child life therapists. 

Patients are treated in the Gambino Medical and Science Foundation Bone Marrow Transplantation Unit, a modern eight-bed transplant unit with rooms large enough to easily allow a parent to be present 24 hours a day. Other personnel who are involved in daily patient care include physical therapists, nutritionists, child psychologists and clinical pharmacists, all of whom coordinate their efforts with those of the attending transplant physician.

Our program remains at the forefront of transplantation research and technology through Institutional Review Board (IRB)-approved, internally initiated studies and research, as well as through affiliations with national and international organizations, including the National Marrow Donor Program (NMDP), the Center for International Blood and Marrow Transplant Research (CIBMTR), the Blood and Marrow Transplant Clinical Trials Network (BMT-CTN) and the Children’s Oncology Group (COG).

If necessary, the team has immediate access to other pediatric subspecialists, including:

• Critical care medicine
• Infectious diseases
• Pulmonology
• Gastroenterology
• Nephrology
• Cardiology
• Neurology
• Ophthalmology
• Dermatology 

• To provide state-of-the-art blood and marrow stem cell transplants
• To promote research in order to improve the results of transplantation
• To improve outcomes of patients diagnosed with malignant and nonmalignant diseases

Our program performs both autologous and allogeneic stem cell transplants. Autologous stem cell transplantation involves a patient using his or her own stem cells. This modality is used predominantly for patients diagnosed with solid tumors, as well as patients with Hodgkin/non-Hodgkin lymphomas.

Allogeneic transplantation

An allogeneic hematopoietic stem cell transplant is a transplant from a donor who is immunologically compatible with the patient and involves matching a patient’s tissue type, specifically their human leukocyte antigen (HLA) type, with that of a related or unrelated donor. While the optimal donor is generally a family member with the same HLA type as the patient, allogeneic transplants may also be performed from unrelated donors (either volunteer donors from a registry, or units of umbilical cord blood). Approximately 70 percent of patients who require an allogeneic transplant have an HLA-identical sibling or unrelated donor available.

For patients who need a stem cell transplant but do not have an HLA-matched related or unrelated donor, recent medical advances have made possible the use of a partially matched, or haploidentical, related donor. A haploidentical-related donor is a 50 percent match to the patient and may be the patient’s parent, sibling or child. Thus, with the advent of partially matched related-donor transplantation, nearly all patients in need of a transplant will have a suitable donor.

The preparative (also called “conditioning”) regiment for transplantation, depending upon the diagnosis and current ongoing trials, will include high-dose, multi-agent chemotherapy, with or without total-body irradiation. For certain disorders (e.g., sickle cell anemia, thalassemia, immunodeficiency syndromes), reduced intensity chemotherapy may be used as the conditioning regimen.     

We perform transplants for the following conditions:

Leukemia/lymphoma

• Acute myeloblastic leukemia (AML), if medically indicated
• Acute lymphoblastic leukemia (ALL), if medically indicated
• Myelodysplastic syndrome (MDS)
• Chronic myelogenous leukemia (CML), if medically indicated
• Juvenile myelomonocytic (JMML) and chronic myelomonocytic (CMML) leukemia

Hemoglobinopathies

• Sickle cell anemia
• Thalassemia major

Bone marrow failure syndromes

• Amegakaryocytic thrombocytopenia
• Aplastic anemia
• Diamond-Blackfan anemia
• Dyskeratosis congenita
• Fanconi anemia
• Severe congenital neutropenia
• Shwachman-Diamond syndrome
• Osteopetrosis
• Other rare bone marrow failure syndromes

Lysosomal and other metabolic storage diseases

• Hurler syndrome
• Metachromatic leukodystrophy
• Adrenoleukodystrophy

Solid tumors

• Neuroblastoma
• Malignant brain tumors
• Other solid tumors as medically indicated

Immunodeficiency disorders

• Severe combined immunodeficiency
• Wiskott-Aldrich syndrome
• Chronic granulatomous disease 

Discharge planning begins on admission and is an integral part of the care we provide. Each patient’s case manager coordinates the services of the interdisciplinary team in preparation for the patient’s transition to the home. Extensive teaching and planning is required before discharge with special attention to home environment, activity limitations, nutrition, rehabilitation, education services and follow-up. Close follow-up treatment usually includes visits to the ambulatory clinic and continued psychosocial supportive and/or educational services.