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Blood and Marrow Transplantation

Our Expertise

The blood and marrow transplant team offers patients the most current treatments available. The pediatric specialists include:

  • Hematologists/oncologists/BMT physicians
  • BMT nurse practitioners
  • BMT nurse coordinator
  • BMT clinical research nurses and associates
  • Registered nurses
  • Transfusion medicine specialists
  • BMT clinical pharmacists
  • Intensivists
  • Surgeons
  • Gastroenterologists and nutritionists
  • Infectious disease specialists
  • Radiation oncologists
  • Oncology/BMT nurse educator

The BMT team develops a comprehensive treatment plan for each patient that includes consultation and evaluation, patient and family education, stem cell donor coordination and procurement, hospitalization details, outpatient visits, home care, school integration, and family support.

Conditions We Treat

Our BMT program offers state-of-the-art, disease-specific treatment protocols for the following conditions:

Leukemias
  • acute lymphoblastic leukemia (ALL)
  • acute non-lymphoblastic leukemia (ANLL)
  • chronic myeloid leukemia (CML)
  • juvenile myelomonocytic leukemia (JMML)
Solid tumors
  • neuroblastoma
  • Wilms tumor
  • sarcomas (Ewings, rhabdomyosarcoma, osteogenic and other soft tissues tumors)
  • desmoplastic small round cell tumor
  • germ cell tumors
  • selected others
Lymphoma
  • Hodgkin's
  • non-Hodgkin's
Central nervous system tumors (brain and spinal cord)
  • medulloblastoma
  • primary neuroectodermal tumor (PNET)
  • astrocytoma
  • ependymoma
  • glioblastoma multiforme (GBM)
  • selected others
Marrow failure
  • aplastic anemia
  • Fanconi's anemia
  • myelodysplastic syndrome (MDS)
  • selected others
Immunological diseases
  • Wiskott-Aldrich syndrome
  • chronic granulomatous disease
  • severe combine immunodeficiency syndrome (SCIDS)
  • hemophagocytosis lymphohistiocytosis (HLH)
  • selected others
Hemoglobin disorders
  • thalassemia
  • sickle cell disease
  • selected others
Histiocytosis
Metabolic genetic disorders
  • galactosylceramide lipidosis (Krabbe's disease)
  • Hurlers syndrome
  • adrenoleukodystrophy (ALD)
  • metachromatic leukodystrophy
  • selected others
Autoimmune diseases
  • juvenile rheumatoid arthritis
  • systemic lupus erythematous
  • scleroderma
  • dermatomyositis/polymyositis
  • multiple sclerosis
  • rheumatoid arthritis
  • selected others

Diagnostic Services

In order to arrive at a precise diagnosis, determine the disease process, and develop an effective treatment plan, a number of tests may be necessary. These include:

  • ultrasound
  • CT scan
  • MRI
  • gallium scan
  • bone scan
  • PET scan
  • bone marrow aspiration and biopsy, and
  • specific blood tests, including tissue typing for compatibility between donor and child
The goal is to verify the proper diagnosis and assign an accurate level of severity of the disease. Often, the evaluation conducted by our BMT team is part of a second opinion and helps families to clarify their treatment options.

Important Treatment Options

The Rationale for BMT
There are several purposes for utilizing blood or marrow stem cells to treat a childs disease. In certain cases of childhood cancer, high dose therapy (chemotherapy and/or radiation therapy) is required to eradicate the remaining cancer cells. This procedure can only be performed by protecting the child's bone marrow from irreversible toxicity through the infusion of blood or marrow stem cells after the therapy.

A second potential use of blood and marrow transplantation is to allow immune cells from another human donor (allogeneic) to destroy the patients remaining cancer cells by cellular immune therapy.

A third use for blood and marrow stem cells is to correct a genetic defect in an existing blood cell, immune cell and/or tissue cell with new, normal cells from another human donor.

Types of Transplants

There are two main types of blood and marrow transplantation:

Autologous (self) transplant uses blood or bone marrow cells from the patients themselves. This form of treatment is mainly used for patients with cancer.

Allogeneic (non-self) transplant uses blood or bone marrow cells from another human being. This type of transplant is used for both cancer and non-malignant conditions, such as immune deficiencies, genetic disorders, and blood disorders.

Stem cells are the cells required for transplant. Stem cells are very primitive or early growing cells that can develop into the mature blood cells that circulate in the blood stream. There are three sources of stem cells for transplantation: bone marrow (center of the bone where blood cells are formed), blood (collected by a process called apheresis) or umbilical cord blood (cells collected from the placenta after delivery of a child).

Stem cells are found not only in bone marrow, but also in the bloodstream and umbilical cord blood. Children needing an allogeneic blood or marrow transplant unfortunately have only a 25 percent chance of finding a good tissue match from a family member. Alternative sources of allogeneic stem cells are mismatched family members, matched unrelated adult donors, and unrelated cord blood donors.

The BMT program has a special program to search for an adult blood donor match from the millions of donors registered with the U.S. National Marrow Donor Program and other associated international BMT donor registries. We coordinate with the registries to collect (or harvest) the stem cells from the donor and safely transport the cells to the hospital for transplantation.

Our program offers the following types of stem cell transplants:

Autologous (self)
  • blood
  • bone marrow
  • cord blood

Allogeneic (non-self)
  • family donor cord blood (matched or mismatched)
  • family donor blood (matched or mismatched)
  • family donor bone marrow (matched or mismatched)
  • unrelated donor cord blood
  • unrelated donor blood
  • unrelated donor bone marrow

Our transfusion medicine staff are specially trained and experienced in the process of apheresis (collecting stem cells) in very young children and infants, both patients and donors.

BMT Treatment Process

After an in-depth review of each childs specific needs, a detailed treatment plan is designed that coordinates all phases of the transplantation process.

The actual infusion of stem cells resembles a typical blood transfusion and takes anywhere from 30 to 90 minutes. However, the process is preceded by careful preparation that includes a critical evaluation of the childs eligibility for BMT and pre-BMT test results, procurement of stem cells from the donor and/or child, and administration of conditioning chemotherapy, immunotherapy and/or radiotherapy.

The weeks following blood or marrow transplant are critical. Pre-transplantation conditioning and preparative therapy disable the patients immune defense system thereby increasing the vulnerability to serious infections. A specially trained team constantly monitors the patient to identify and counteract complications or side effects.

To help reduce the risk of the patient developing serious infection, special precautions are taken to reduce exposure to viral, bacterial, or fungal infections, along with the administration of several antibiotics after transplantation. The patient recovers in an isolation facility equipped with special unidirectional positive-pressure airflow.

Following transplantation, caregiving teams track the progress of stem cells as they begin to migrate to the cavities of the large bones and produce normal blood cells, a process known as engraftment. Other organ functions (heart, lung, liver, etc.) are also carefully monitored. For all allogeneic transplants, the percentage of donor-originated blood cells are measured against the patients own surviving cells. When an appropriate ratio is reached, the child is gradually taken off antibiotics, and blood and platelet transfusions are reduced. When a sufficient number of healthy red blood cells, white blood cells, and platelets are produced, the patient is ready to go home.

Following discharge, the programs physicians and nurses continue to closely follow the progress of the child. The BMT team arranges home care, lab work and therapies, if needed, and administers medications, as required. During their outpatient visits, children are visited by music and pet therapists, which helps them cope with the stresses of treatment process and associated psychological issues.

Contact

Pediatric Blood and Marrow Transplantation
Directions
(212) 305-8316
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