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BMT Research

The physicians at Children’s BMT program are constantly striving to improve cellular therapies for treatment of childhood cancer and blood diseases. A number of studies are underway looking at treatment methods that will make marrow and cord blood transplants kinder, gentler and safer. Their ultimate goals are to reduce the complication rate and improve quality of life for children undergoing this procedure.

Meet the Researchers

Research Interests

Graft-versus-Host Disease

Dr. Rakesh Goyal’s clinical research is focused on understanding the relationship between individual genetic variability and the outcome of transplant therapy. He and his team are studying ways we may be able to predict the risk of serious complications, such as graft-versus-host disease, and how that might lead to individualized therapy.

Read more about Dr. Goyal’s research on Graft-Versus-Host Disease.

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Ex-vivo Stem Cell Expansion

The use of umbilical cord blood in transplant is another area of focus at Children’s Hospital of Pittsburgh. Cord blood is used the same way bone marrow is in transplant, except that a perfect match is not necessary for cord blood transplantation to attain the same positive outcome.

Dr. Peter Shaw’s area of research is ex-vivo stem cell expansion. He is manipulating cord blood stem cells in an attempt to shorten the time it takes for platelets in cord blood to find a home in the recipient’s marrow.

Read more about Dr. Shaw’s research on ex-vivo stem cell expansion.

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BMT for Sickle Cell Disease and Thalassemia

Dr. Lakshmanan Krishnamurti’s clinical research is focused on developing newer, gentler types of bone marrow transplantation for patients with inherited blood disorders, such as sickle cell disease and Thalassemia. Most often, stem cell transplantation is used to treat cancer. However, BMT is the only procedure that can cure sickle cell disease, a blood disorder affecting more than 50,000 Americans.

Read more about Dr. Krishnamurti’s research on BMT for Sickle Cell Disease and Thalassemia.

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Clinical Trials

Protocol Type of Trial Sponsorship*

Reduced Intensity Conditioning Hematopoietic Cell Transplantation for Pediatric Patients with Hematologic Malignancies at High Risk for Transplant Related Mortality with Standard Transplantation; A Multi-center Trial for the Pediatric Blood and Marrow Transplant Consortium (PBMTC)

Treatment

PBMTC

A Pilot Study of Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Non-Myeloablative Preparative Regimen to Achieve Stable Mixed Chimerism

Treatment

Institutional

Rapamycin for Immunosuppression and B Cell Modulation Post Stem Cell Transplant for Acute Lymphoblastic leukemia

Treatment

Limited Institution Pilot

ANBL0032, Phase III Randomized Study of Chimeric Anti-GD2 in High Risk Neuroblastoma Following Myeloablative Therapy and Autologous Stem Cell Rescue

Treatment

COG

Initial Systemic Treatment of Acute GVHD: A Phase II Randomized Trial Evaluation Etancercept, Mycophenolate Mofetil (MMF), Denieukin Difitox (Ontak) and Pentostatin in Addition to Corticosteriods

Treatment

BMT CTN

National Marrow Donor Program (NMDP) Recipient Consent for Participation in Research Database and/or Research Sample Respository

Biology

NMDP

Defibrotide for the Treatment of Venoocclusive Disease of the Liver

Treatment

Institutional

GVH0112, Cytokine Gene Polymorphisms in Pediatric Patients Receiving Tacrolimus or Cyclosporin for Graft-versus-Host Disease Prophylaxis

Biology

PBMTC

PBMTC051: Soluble Tumor Necrosis Factor Receptor Embrel (Etanercept) for the Treatment of Acute Non-Infectious Pulmonary Dysfunction (Idiopathic Pneumonia Syndrome following Allogeneic Stem Cell Transplantation

Treatment

COG

For more information about trial participation, please call 412-692-6740.

* Sponsorship
COG = Children’s Oncology Group
NMDP = National Marrow Donor Program
PBMTC = Pediatric Blood & Marrow Transplant Consortium
BMT CTN = Blood and Marrow Transplant Clinical Trials Network

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Last Update
November 9, 2011
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Last Update
November 9, 2011
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