Haploidentical Donor BMT Program Launched

New hope for patients with no bone marrow donor match

Blood cancers, such as leukemia, and other blood diseases, such as sickle cell anemia, are potentially curable with a blood and marrow transplant, but, until recently, only two-thirds of patients were able to find a suitably matched donor. The remaining third with no donors were not able to get the benefit of transplant.

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Traditionally, an HLA-matched donor is preferred for blood and marrow transplantation. In April 2014, Cleveland Clinic launched a haploidentical donor transplantation program that now allows use of HLA-half matched (haplotype) donors. Biological parents and children and half-matched siblings can serve as donors. The ability to use haploidentical donors is of special relevance to patient populations who previously had very limited sibling and unrelated donor options, such as patients with sickle-cell disease that disproportionately afflicts African-Americans.

“This is a step toward offering a donor for every patient,” says Rabi Hanna, MD, Director, Pediatric Blood & Marrow Transplant Program, Cleveland Clinic Children’s. “Now, barring any health conditions or other issues that would prevent donation, a parent can donate and siblings may also be half matches.”

The preferred donor sources continue to be an HLA-identical sibling or HLA-matched unrelated donor. For patients without these donor options, a haploidentical donor or umbilical cord blood can be used for transplantation.

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“We can add haploidentical donor transplantation to the list of significant advances in transplant technology and supportive care that have positively impacted patient survival and outcomes,” says Navneet Majhail, MD, MS, Director, Adult Blood & Marrow Transplant Program, Cleveland Clinic. “With a donor option now available for more or less all patients, timely referral for a transplant is key to achieving optimal patient outcomes.”

Seven procedures have been performed as of July, with a 30-day survival rate of 100 percent.

Overcoming Rejection

The concept of haploidentical transplantation is not new, but previous technologies have been thwarted by high rates of graft rejection or a potentially fatal post-transplant complication called graft-versus-host disease. To overcome these significant risks, the new approach involves administering cyclophosphamide in the immediate days following transplant to destroy alloreactive immune cells that mediate the graft-versus-host and host-versus-graft reactions while sparing hematopoietic stem cells. High rates of engraftment with acceptable risks of transplant complications and mortality have been reported with this technique.

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Serving Children and Adults

Cleveland Clinic is among the few programs in the nation to routinely offer haploidentical donor transplantation, and serves adult and pediatric patients. Haploidentical transplantation is also available for patients who qualify for participation in a large NIH clinical trial, which is also available at Cleveland Clinic.