Blood and Marrow Clinical Trials Network (BMT CTN) Bone Marrow Transplant From Partially Matched Donors and Nonmyeloablative Conditioning for Blood Cancers (0603)

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Accession Number

Study Type
Clinical Trial

Collection Type
Open BioLINCC Study See bottom of this webpage for request information

Study Period
October 2008 to November 2013

NHLBI Division

Dataset(s) Last Updated
January 3, 2018

Related Studies
BMT CTN-0604


Commercial Use Data Restrictions No

Data Restrictions Based On Area Of Research No


Using the reduced intensity conditioning (RIC) transplant technique, this study evaluated survivorship of recipients who received a bone marrow transplant from a related and partially matched donor.


Individuals with aggressive forms of the blood cancers leukemia and lymphoma typically receive targeted therapy, chemotherapy, and radiation as part of their treatment options. When these and other therapies have been exhausted, it is recommended that the patient receive a bone marrow or cord blood transplant. A first degree relative, if available, is likely to be the best candidate as a bone marrow donor if the human leukocyte antigen (HLA) markers are a sufficient match.

For recipients who find a suitable match, high doses of chemotherapy are administered preemptively in an attempt to reduce the likelihood of transplant rejection or graft-versus-host disease (GVHD). Often though, individuals greater than 60 years of age or with multiple medical conditions are disqualified from the regimen due to their inability to withstand the chemotherapeutic agents’ toxicity levels.

The BMT CTN 0603, or Haplo-marrow trial, researched the effectiveness of a type of bone marrow transplant called reduced intensity conditioning (RIC) or nonmyeloablative transplant for individuals who were only a partial or haploidentical match with a family member. Specifically, recipients were given lower doses of chemotherapy prior to receiving the transplant and also received a smaller quantity of transplanted bone marrow than is typically administered.

The BMT CTN 0603 study was one of two parallel multicenter phase 2 studies; the other of which was BMT CTN 0604, which used double umbilical cord blood transplantation. The goal of these studies was to generate pilot multicenter data to support a future phase 3 randomized clinical trial.


A total of 50 individuals participated in and completed the BMT CTN 0603 study. Eligibility criteria included a diagnosis of acute leukemia or lymphoma and an age of less than 70 years. Those less than 22 years, were potentially eligible if they were ineligible for the BMT CTN #0501 trial. Participants were also required to have a partial tissue match with a first-degree family member who was at least 18 years of age. Other inclusion criteria included a left ventricular ejection fraction of at least 35% and specific liver, kidney, pulmonary, and physical functioning scores within acceptable ranges.

Those who were pregnant, breastfeeding, HIV positive or had a suitable donor were excluded.


The study consisted of 2 parallel, multicenter trials where reduced intensity conditioning (RIC) was used as a transplant procedure in each trial. In the BMT CTN 0603 study, HLA-haploidentical (Haplo-marrow) bone marrow was transplanted into recipients from a related donor. For the BMT CTN 0604 study, double umbilical cord blood (dUCB) was transplanted in unrelated recipients.

As part of the RIC procedure in the BMT CTN 0603 study, participants were given two chemotherapeutic agents and radiation prior to receiving a bone marrow transplant. Fludarabine, 30 mg/m2 was given on five occasions and cyclophosphamide (Cy) 14.5mg/kg was given on two occasions; both administered intravenously. One day prior to the transplant, participants were also administered 200 centigray (cGy) total body irradiation. Following the transplant, participants received several agents to offset GVHD which included: Cy, tacrolimus, mycophenolate mofetil (MMF), and granulocyte-colony stimulating factor (filgrastim). At the discretion of the treating physician, participants could also receive supportive care such as blood transfusions, prophylaxis, and therapy to treat infections.

The primary outcome for both trials was participant survival from the time of transplant to 180 days measured at 6 and 12 month intervals. There were several secondary outcomes including neutrophil and platelet recovery times, treatment related mortality, and primary graft failure.


Of the 50 participants in the BMT CTN 0603 study, 83.7% survived to the 6-month benchmark and 62.0% survived to the 1-year benchmark. Neutrophil recovery at day +56 was 96%, platelet recovery at day +100 was 98%, treatment related mortality was 4% at 6 months, and there was one case of primary graft failure.

Outcomes from both clinical trials suggest that the rates of survivorship are comparable to those where there was a matched unrelated donor.

Brunstein CG, Fuchs EJ, Carter SL, et al. Alternative donor transplantation after reduced intensity conditioning: results of parallel phase 2 trials using partially HLA-mismatched related bone marrow or unrelated double umbilical cord blood grafts. Blood. 2011;118(2):282-288. doi:10.1182/blood-2011-03-344853.

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