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What is a Stem Cell Transplant?

Standard of Care Protocols

Investigational Protocols


What is a Stem Cell Transplant?

How stem cells are obtained

Stem cells used for transplantation can be obtained from bone marrow, umbilical cord blood, or mobilized peripheral blood. Stem cells can be obtained from the bone marrow in the operating room under general anesthesia or from the peripheral blood by a process called apheresis, where the stem cells are collected and the rest of the blood is then returned to the donor. Cord blood is collected at the time of delivery from volunteer mothers and frozen until it is used.

Allogeneic stem cells

Allogeneic stem cells are collected from a related or unrelated donor. The allogeneic donor is chosen to be as genetically close to the patient as possible. The best donor is a brother or sister who has the same HLA antigens as the patient. Siblings have a 25 percent chance of being such a donor. If a brother or sister cannot be a donor, we contact the National Marrow Donor Program (NMDP) to see if an unrelated volunteer donor has HLA typing similar to that of the patient, and therefore, could be used as a stem cell donor. In cases when an unrelated donor cannot be identified, the father or mother (who have 50 percent of the same genes as the patient) can be used in a haploidentical transplant or unrelated cord blood can be used as a source of stem cells.

Autologous stem cells

For some forms of cancer, the patient’s own stem cells can be collected from the bone marrow or peripheral blood and used for transplant.

Pre-transplant therapy

Before transplantation, patients receive therapy to destroy the cancer or abnormal stem cells and  allow normal stem cells to grow. The specific therapy that is administered will depend upon the patient’s disease and clinical history.

Hospital stay

After the completion of pre-transplant therapy, the stem cells are infused into the patient’s vein where they can travel to the patient’s bone marrow. The patient must remain in the hospital's Stem Cell Transplant Unit until the donor stem cells have grown enough to permit the patient to safely leave the hospital, usually 1 to 2 months after the transplant. 

Standard of Care Protocols

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Patients with oncological, immunological, hematological and metabolic diseases are candidates for hematopoietic stem cell transplantation. Decisions regarding the specific stem cell source used for transplantation, the specific donor and the recommended pre-transplant therapy are dependent upon multiple factors such as the patient’s age, disease, and status, prior therapy and clinical history, including prior infections.

Leukemia

Treatment Plan 9142A, 9022A - Acute Lymphoblastic Leukemia
Patients younger than 3 years of age who have matched related or unrelated donors will receive bone marrow or peripheral blood stem cells after pre-transplant therapy with cyclophosphamide and busulphan. 

Treatment plan 9142B, 9022B - Acute Lymphoblastic Leukemia
Patients older than 3 years of age who have matched related donor or unrelated donors will receive bone marrow or peripheral blood stem cells after pre-transplant therapy with total body radiation and etoposide or cyclophosphamide. 

Treatment Plan 9022A - Acute Myeloid Leukemia, Myelodysplastic Syndrome
Patients who have matched related or unrelated donors will receive bone marrow or peripheral blood stem cells after pre-transplant therapy with cyclophosphamide and busulphan. For patients who have mismatched related or unrelated donors, a Phase 1 trial is open that uses conditioning with total body irradiation and cyclophosphamide (Protocol 297).

Treatment Plan 9905B - Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Juvenile Myelomonocytic Leukemia
Patients younger than 3 years of age with acute myeloid leukemia, acute lymphocytic leukemia, chronic myeloid leukemia or juvenile myelomonocytic leukemia who have related or unrelated cord blood will be transplanted after pre-transplant therapy with cyclophosphamide, busulfan and fludarabine.

Treatment Plan 9905A - Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Myeloid Leukemia, Juvenile Myelomonocytic Leukemia
Patients older than 3 years of age who have related or unrelated cord blood will be transplanted after pre-transplant therapy with cyclophosphamide, fractionated total body irradiation and fludarabine.

Treatment Plan 9926 - Acute Myeloid Leukemia, Myelodysplastic Syndrome
Patients with acute myeloid leukemia or myelodysplastic syndrome who have matched related or unrelated donors, will be transplanted with bone marrow or peripheral blood stem cells after pre-transplant therapy with busulfan, cyclophosphamide and thymoglobulin.

Lymphoma

Patients with Hodgkin’s disease or non-Hodgkin’s lymphoma beyond first remission will be transplanted with autologous peripheral blood stem cells. The decision about the appropriate pre-transplant therapy will be made after reviewing their prior chemotherapy.

Treatment Plan 9024
Patients with Hodgkin’s disease or non-Hodgkin’s lymphoma will receive autologous peripheral blood stem cells after pre-transplant therapy with cyclophosphamide, etoposide and carmustine.

Treatment Plan 9135
Patients with Hodgkin’s disease or non-Hodgkin’s lymphoma will receive autologous peripheral blood stem cells after pre-transplant therapy with cyclophosphamide, etoposide, carmustine, gemcitabine and vinorelbine.

Neuroblastoma

Patients with Neuroblastoma or ganglioneuroblastoma will be transplanted with autologous peripheral blood stem cells. The decision about the appropriate pre-transplant therapy will be made after reviewing their prior chemotherapy.

Treatment Plan 9145
Patients with neuroblastoma or ganglioneuroblastoma will receive autologous peripheral blood stem cells after pre-transplant therapy with etoposide, melphalan and carboplatin.

Treatment Plan 9198
Patients with neuroblastoma or ganglioneuroblastoma will receive tandem transplants with autologous peripheral blood stem cells. Pre-transplant therapy for the first transplant will be cyclophosphamide and etoposide and pre-transplant therapy for the second transplant will be ThioTEPA and melphalan.

Treatment Plan 9925
Patients with neuroblastoma will receive autologous peripheral blood stem cells with no pre-transplant conditioning treatment. Autologous peripheral blood stem cells are infused to prevent marrow suppression after metaiodobenzylguanidine.

Solid Tumors

Patients with Ewing’s sarcoma, Wilms' tumors; rhabdomyosarcoma, neuroblastoma, germ cell tumors, and other soft tissue sarcomas will be transplanted with autologous peripheral blood stem cells. The decision about the appropriate pre-transplant therapy will be made after reviewing their prior chemotherapy.

Treatment Plan 9918:
Patients with Ewing’s sarcoma, Wilms tumors, rhabdomyosarcoma, neuroblastoma; germ cell tumors; and other soft tissue sarcomas will be transplanted with autologous bone marrow or peripheral blood stem cells after pre-transplant treatment with buslufan, melphalan and topotecan.

Aplastic Anemia

Treatment Plan 9018
Patients with severe aplastic anemia who have a related matched donor will receive bone marrow or peripheral blood stem cells after pre-transplant therapy with cyclophosphamide and thymoglobulin.

Treatment Plan 9914
Patients with severe aplastic anemia who have a related mismatched donor or an unrelated matched donor will receive bone marrow, peripheral blood stem cells or cord blood after pre-transplant treatment with cyclophosphamide, thymoglobulin, low dose fractionated total body irradiation and fludarabine.

Treatment Plan 9920
Patients with Fanconi anemia who have a related matched donor will receive bone marrow or cord blood stem cells after pre-transplant therapy with modified doses of cyclophosphamide, thymoglobulin and fludarabine.

Treatment plan 9922
Patients with Fanconi anemia, who have an unrelated donor, will receive bone marrow stem cells after pre-transplant therapy with modified doses of cyclophosphamide, thymoglobulin and fludarabine and single low dose of total body irradiation. The T cell content of the unrelated donor cells is reduced by graft manipulation to prevent graft versus host disease.

Immunological, Hematological and Metabolic Genetic Diseases

Treatment Plan 9187
Patients with lethal or sublethal genetic lymphohematological diseases who have related matched donors will be transplanted with bone marrow, peripheral blood stem cell or cord blood after pre-transplant therapy with a reduced intensity regimen containing cyclophosphamide, busulfan, fludarabine and alemtuzumab.

Treatment Plan 9905

Patients with genetic diseases including Mucopolysaccharidosis I (Hurler’s Syndrome), Wiskott–-Aldrich syndrome, other non-SCID immune odeficiencies,; Diamond– Blackfan anemia,; refractory Langerhans cell histiocytosis, and hemophagocytic lymphohistiocytosis, who have cord blood donors, will receive pre-transplant therapy with cyclophosphamide, busulfan and fludarabine.

Treatment Plan 9912
Patients with genetic diseases including metabolic disorders (Hurler’s Syndrome, adrenoleukodystrophy), and immunodeficiencies (Wiskott-Aldrich syndrome and other non-SCID immune deficiencies), who have related or unrelated donors, will be transplanted with bone marrow or peripheral blood stem cells after pre-transplant therapy with cyclophosphamide, busulfan and thymoglobulin.

Investigational Protocols

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In spite of recent advances, Hematopoietic Stem Cell Treatment (HSCT) patients may have severe complications. The investigational protocols of the Pediatric Stem Cell Transplant program addresses some of these complications, including chemotherapy-induced side effects, graft versus host disease and infections.

A Phase 1 study to evaluate the safety and tolerability of tandemly purified allogeneic CD34+ CD90+ hematopoietic stem cells (HSC) administered following conditioning with AMG 191 to achieve engraftment and immune reconstitution in patients with severe combined immunodeficiency (SCID) — Principal investigator: Rajni Agrawal, MD (Protocol 300)
HSC engraftment requires the reduction or elimination of the host HSC by the administration of irradiation and/or chemotherapy, both of which have significant side effects. To explore whether it is possible to achieve donor HSC engraftment without the use of either irradiation or chemotherapy, patients on this protocol will receive a single dose of a monoclonal antibody to c-kit (CD117) followed by the infusion of the donor CD34 positive cells. The first patients to be treated are patients originally diagnosed with SCID who received a HSC transplant without chemotherapy and who have no evidence of donor HSC engraftment. Patients will receive increasing amounts of antibody until donor HSC engraftment is achieved. Subsequent patients will then be transplanted with purified HSC rather than CD34 positive cells. If successful, the use of the anti-CD117 antibody to achieve HSC engraftment will reduce the toxicities associated with HSCT.

Use of T-allo10 cell infusions combined with mismatched related or unrelated donor hematopoietic stem cell transplantation (HSCT) for hematological malignancies — Principal investigator: Rajni Agrawal, MD (Protocol 297)
HSCT with mismatched donors frequently results in severe and sometimes fatal acute graft versus host disease. The administration of regulatory T cells can prevent acute graft versus host disease. T-allo10 cells are donor-derived regulatory T cells that have been made specifically tolerant to the patient. The T-allo10 cells will be infused on the day prior to HSCT. Patients will receive increasing numbers of the T-allo10 cells and will be followed to see how long the T-allo10 cells survive and if there are any side effects to their administration. If successful, the administration of the T-allo10 cells will prevent acute graft versus host disease in a high-risk population and thus improve the overall results of HSCT.

Phase I/II study of CaspaCide T cells from an HLA partially matched related donor after negative selection of TCR aß+T and B  cells in pediatric patients— Principal investigator: Rajni Agrawal, MD (Protocol 307)
Patients with mismatched family donors will be transplanted with peripheral blood stem cells from which the aß+T and B cells have been removed.  To improve their immune reconstitution after HSCT, they will be infused with donor T cells that have been modified so that they can be eliminated if severe graft versus host disease occurs. If successful, the combination of T/B cell removal plus the addition of the modified T cells will result in no graft versus host disease and improved immune reconstitution.

An open-label, randomized, adaptive, two-arm, multicenter trial to evaluate pharmacokinetics and pharmacodynamics of two doses of oseltamivir phosphate (Tamiflu) in the treatment of influenza in immunocompromised children younger than 13 years of age with confirmed influenza infection — Principal investigator: Ami Shah, MD (Protocol 305)
Because HSCT patients have poor immune systems, common infections like influenza can be dangerous and sometimes fatal. The protocol will measure the drug levels in HSCT patients with influenza infections who are receiving oseltamivir (Tamiflu) as therapy to determine the appropriate dose in pediatric HSCT patients.

Protocol for hematopoietic stem cell transplantation (HSCT) for treatment of symptomatic genetic lymphohematological diseases for patients lacking a fully HLA-matched sibling donor — Principal investigators: Ken Weinberg, MD, and Sandeep Soni, MD (Protocol 246) 
The standard chemotherapy given as preparation for HSCT for patients with genetic diseases who do not have histocompatible sibling donors contains high doses of cyclophosphamide (Cytoxan), which can result in significant side effects. To reduce the side effects associated with HSCT in these patients, the dose of cyclophosphamide will be reduced to determine whether HSC engraftment can be achieved with fewer side effects.

A randomized, double-blind Phase 3 study of ibrutinib in combination with corticosteroids versus placebo in combination with corticosteroids in subjects with new onset chronic graft-versus-host disease (cGVHD) — Principal investigator: Ami Shah, MD (Protocol 315)
Chronic graft versus host disease continues to be a severe complication of allogeneic HSCT. Ibrutinib has shown promise for the treatment of chronic graft versus host disease. In this protocol, HSCT patients who have newly diagnosed chronic graft versus host disease will be randomized to receive either

Multicentered, open-label randomized study of single or double myeloablative cord blood stem cell transplantation with or without infusion of off-the-shelf ex vivo expanded cryopreserved cord blood progenitor cells in patients with hematologic malignancies — Principal investigator: Rajni Agarwal, MD (Protocol 371)
Patients receiving HSC transplants with unrelated cord blood units can have delays in hematopoietic engraftment compared to patients receiving bone marrow or peripheral blood HSC. In an attempt to hasten hematopoietic recovery after unrelated cord blood transplantation, patients in a randomized Phase 3 clinical trial may receive third-party cord blood cells following transplantation, that have been cultured with notch ligand and which, in preliminary clinical trials, have resulted in more rapid hematopoietic recovery. 

Protocol for related donor hematopoietic stem cell transplantation (HSCT) for treatment of symptomatic genetic lymphohematological diseases; principal investigators, Ken Weinberg/Sandeep Soni, M.D. (Protocol 294)
Many genetic diseases involving both the immune and hematopoietic systems can be cured by the transplantation of HSC from histocompatible, unaffected siblings.  However, toxicities related to the chemotherapy given prior to transplantation can occur, especially related to the administration of cyclophosphamide (Cytoxan). The purpose of the protocol is to determine whether the successful engraftment of normal sibling HSC can occur when the patient receives fludarabine instead of cyclophosphamide prior to transplantation.

An intermediate-size, expanded access protocol to provide brincidofovir for the treatment of serious adenovirus infection or disease; principal investigator, Rajni Agarwal, M.D. (Protocol 301)
Adenovirus infections can be severe and sometimes fatal in pediatric HSCT patients. The standard treatment for adenoviral infections is cidofovir, which has significant renal side effects. Brincidofovir is a liposomal form of cidofovir with less renal side effects. The protocol is to evaluate the benefits and side effects of Brincidofovir in HSCT patients who have adenoviral infections after transplantation.

A phase 3, randomized, adaptive study comparing the efficacy and safety of defibrotide vs. best supportive care in the prevention of hepatic veno-occlusive disease in adult and pediatric patients undergoing hematopoietic stem cell transplant; principal investigator, Rajni Agrawal, M.D. (Protocol 314)
Veno-occlusive disease is a severe complication that can occur after HSCT. Defibrotide has been shown to positively impact the course of HSCT patients with clinical veno-occlusive disease. This protocol will compare veno-occlusive disease in pediatric HSCT patients who receive defibrotide to prevent the development of veno-occlusive disease to patients who receive best supportive care. The frequency and severity of veno-occlusive disease in the two groups will be compared.

Pilot safety and feasibility trial of sirolimus and mycophenolate for prevention of GvHD in mismatched unrelated and related donor hematopoietic stem cell transplantation for hematologic malignancies; principal investigator, Rajni Agarwal, M.D. (Protocol 295)
Graft versus disease remains a major complication of HSCT in spite of the routine prophylactic use of cyclosporine/tacrolimus plus other drugs to prevent/reduce graft versus host disease. The protocol explores the use of a new prophylactic combination, sirolomus plus mycophenolate mofetil (MMF), to prevent graft versus host disease, which may have fewer side effects.

An observational study to characterize safety and efficacy of allogeneic hematopoietic stem cell transplantation for the treatment of childhood cerebral adrenoleukodystrophy; principal investigator, Ami Shah, M.D. (Protocol 279)
Cerebral adrenoleukodystrophy is a severe degenerative neurological disease. Affected patients have been treated by HSCT. This protocol will collect clinical data associated with HSCT for cerebral adrenoleukodystrophy.

Humanitarian use of the CliniMACS device; principal investigator, Rajni Agrawal, M.D (Protocol 309)
The CliniMACS device can isolate CD34 positive cells from an HSC product to reduce the number of T cells that are infused at the time of HSCT thus reducing the frequency and severity of graft versus host disease. The protocol isolates donor CD34 positive cells to be administered to HSCT patients who have poor hematopoietic function after a first HSCT with a reduced risk of acute graft versus host disease.

Clinical Trials

Find more research or connect with researchers on the National Institutes of Health's clinical trials.

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