Choice of conditioning regimens for bone marrow transplantation in severe aplastic anemia

Journal Name
Blood Advances
Primary Author
Bejanyan N
Author(s)
Bejanyan N, Kim S, Hebert KM, Kekre N, Abdel-Azim H, Ahmed I, Aljurf M, Badawy SM, Beitinjaneh A, Boelens JJ, Diaz MA, Dvorak CC, Gadalla S, Gajewski J, Gale RP, Ganguly S, Gennery AR, George B, Gergis U, Gómez-Almaguer D
Vicent MG, Hashem H, Kamble RT, Kasow KA, Lazarus HM, Mathews V, Orchard PJ, Pulsipher M, Ringden O, Schultz K, Teira P, Woolfrey AE, Saldaña BD, Savani B, Winiarski J, Yared J, Weisdorf DJ, Antin JH, Eapen M
Original Publication Date
Diseases

Allogeneic bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. transplantation (BMT) is curative therapy for the treatment of patients with severe aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… (SAA). However, several conditioning regimens can be used for BMT. We evaluated transplant conditioning regimens for BMT in SAA after HLA: See human leukocyte antigen. -matched sibling and unrelated donor: A donor that is not a sibling or other familial relation of the patient (recipient). BMT. For recipients of HLA-matched sibling donor transplantation (n = 955), fludarabine: The U.S. Food and Drug Administration (FDA) approved fludarabine for treating chronic lymphocytic leukemia (CCL). Researchers are studying fludarabine in combination with other medicines for treating bone marrow failure. (Flu)/cyclophosphamide (Cy)/antithymocyte globulin (ATG) or Cy/ATG led to the best survival. The 5-year probabilities of survival with Flu/Cy/ATG, Cy/ATG, Cy ± Flu, and busulfan/Cy were 91%, 91%, 80%, and 84%, respectively (P = .001). For recipients of 8/8 and 7/8 HLA allele-matched unrelated donor transplantation (n = 409), there were no differences in survival between regimens. The 5-year probabilities of survival with Cy/ATG/total body irradiation 200 cGy, Flu/Cy/ATG/total body irradiation 200 cGy, Flu/Cy/ATG, and Cy/ATG were 77%, 80%, 75%, and 72%, respectively (P = .61). Rabbit-derived ATG compared with equine-derived ATG was associated with a lower risk of grade II to IV acute graft-versus-host disease (GVHD): Also called GVHD, it is a common complication of bone marrow/stem cell transplantation. It is caused when the donor's immune cells, now in the patient, begin to see the the patient's body as foreign and mount an immune response. GVHD most commonly effects the recipient's skin, intestines, or liver… (hazard ratio [HR], 0.39; P < .001) but not chronic GVHD. Independent of conditioning regimen, survival was lower in patients aged >30 years after HLA-matched sibling (HR, 2.74; P < .001) or unrelated donor (HR, 1.98; P = .001) transplantation. These data support Flu/Cy/ATG and Cy/ATG as optimal regimens for HLA-matched sibling BMT. Although survival after an unrelated donor BMT did not differ between regimens, use of rabbit-derived ATG may be preferred because of lower risks of acute GVHD.

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