Our Services

About Blood and Marrow Transplantation (BMT) and Cellular Therapies

Description of Services

Our Mission

The clinical mission of the Division of Blood and Marrow Transplantation and Cellular Therapies is to design and test disease-specific and biologically rational novel reduced-toxicity transplantation regimens for patients with high-risk leukemia or lymphoma, and for those afflicted with life-threatening inherited conditions that can lead to bone marrow failure, immune deficiency, autoimmune diseases, and neurodegenerative conditions including but not limited to leukodystrophies and mucopolysaccharidosis syndromes.

Our translational research mission focuses on developing new cellular immune therapy programs for cancer and viral infections and testing the use of bone marrow transplantation following solid organ transplant to establish immunity, tolerance, and extend organ survival.

Blood and marrow transplantation (BMT) is used successfully to treat a number of cancers, blood diseases and immune disorders that were once considered incurable. Our proactive patient care with close monitoring of the immune system has resulted in unsurpassed outcomes since 2010: one-year transplant-related mortality below 10 percent for all allogeneic marrow and cord blood transplants and below 5 percent for reduced-intensity transplants.

The division has a strong focus on reduced-intensity/toxicity cord blood, bone marrow, and stem cell transplantation for inherited disorders (see below). In addition, it has a clinical research focus on Crohn's disease and other autoimmune disorders. In collaboration with other divisions, it also offers tandem lung and bone marrow transplantation for patients with immunodeficiencies who have progressed to pulmonary failure.

Children's Hospital's Program for the Study of Neurodevelopment in Rare Disorders (NDRD) collaborates with the division on Inborn Errors of Metabolism including:

Mucopolysaccharidoses

  • Hurler syndrome (MPS I)
  • Hunter syndrome (MPS II)                  
  • Sanfilippo syndrome (MPS III)

Leukodystrophies

  • Krabbe disease, also known as globoid cell leukodystrophy 
  • Metachromatic leukodystrophy (MLD)
  • X-linked adrenoleukodystrophy (ALD) 
Other inherited metabolic disorders
Alpha mannosidosis
 
Primary immunodeficiency syndromes including but not limited to:
  • Severe combined immune deficiency (SCID)
  • Omenn syndrome
  • Bare lymphocyte syndrome (BLS)
  • Combined immune deficiency (CID) syndromes
  • Combined variable immune deficiency (CVID) syndrome
  • Wiskott-Aldrich syndrome
  • Leukocyte adhesion deficiency
  • Chronic granulomatous disease (CGD)
  • X-linked Hyper IgM (XHIM) syndrome
  • IPEX syndrome
  • Chediak–Higashi syndrome
  • Autoimmune lymphoproliferative syndrome (ALPS)
  • Hemophagocytic lymphohistiocytosis (HLH) syndromes
  • Lymphocyte signaling defects
Bone marrow failure syndromes
  • Aplastic anemia (AA)
  • Fanconi anemia (FA)
  • Schwachman Diamond syndrome
  • Dyskeratosis congenita (DC)
  • Congenital amegakaryocytic thrombocytopenia (CAMT)
  • Osteopetrosis
Hereditary anemias 
  • Thalassemia major
  • Sickle cell disease (SCD)
  • Diamond Blackfan anemia (DBA)

Children’s is also committed to clinical investigation with programs for innovative therapies against malignant and non-malignant diseases. The nursing staff, with training in BMT, oncology and pediatric intensive care, is complemented by pharmacists, social workers, child life specialists, nutritionists and physical therapists, all specializing in the care and treatment of children and adolescents.

The Blood and Marrow Transplant program at Children's is accredited by the Foundation for the Accreditation of Cellular Therapy (FACT) for all components of both allogeneic and autologous transplants, which include the clinical program and stem cell collection. This accreditation certifies that Children’s program has passed rigorous standards of practice for all of these facilities.

To provide our patients with access to the most recent innovations in transplantation, we collaborate in many multi-center trials sponsored by the National Institutes of Health, the Pediatric Blood and Marrow Transplant Consortium and the Children’s Oncology Group (COG). For patients with sickle cell disease and other non-malignant conditions, we are doing pioneering clinical research in a form of transplantation called non-myeloablative, meaning the recipient’s bone marrow is not completely destroyed. We work in close collaboration with the Stem Cell Program at the University of Pittsburgh Cancer Institute (UPCI) .

The Blood and Marrow Transplantation (BMT) program has expanded considerably since its inception in 1991. It is accredited by the National Marrow Donor Program (NMDP) for unrelated donor transplants in children. Transplants performed here utilize bone marrow, peripheral blood, and cord blood as stem cell sources. For patients requiring transplantation who do not have related donors, we have many years of experience with unrelated bone marrow and cord blood transplantation. We routinely perform transplants for patients with a wide variety of malignant (acute and chronic leukemias, brain tumors) and non-malignant (aplastic anemia, sickle cell disease, immune deficiencies) diseases. Types of transplants performed at Children’s include:

 
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Last Update
December 16, 2014
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