SAA is a rare but serious blood disorder. Children and adults with SAA get very sick with low blood counts, infections or bleeding.
If SAA does not respond to the first choice of therapy or comes back (relapses) after a period of health, then we call this relapsed and refractory SAA. Blood or marrow transplant (BMT) is the only cure for relapsed and refractory SAA.
Family members or unrelated volunteers can donate cells if they match with the patient. Many people do not have a fully matched donor, though, to have a BMT. A new study shows that a haploidentical, or half-matched, BMT from a family member can help people live longer.
This especially helps people who are American Indian or Alaska native, Asian, Black or African American, Native Hawaiian, other Pacific Islander, more than one race, or Hispanic. It’s easier for people in these communities to find a related half-matched than a fully matched unrelated BMT donor.
For this study, 31 people with relapsed and refractory SAA got half-matched transplants. People had a variety of racial and ethnic backgrounds: Less than half of the people in this trial were white.
A year after BMT, more than 80% of people were alive. Few people had chronic graft-versus-host disease (GVHD), a possible side effect of BMT. People’s health-related quality of life was normal.
Researchers said 3 things helped control SAA. First, use enough bone marrow cells from the donor for the transplant itself. Second, reducedintensity preparations are enough chemo prior to the BMT. Third, cyclophosphamide reduces the risk of GVHD enough to use a half-matched donor.