Mark C. Walters, University of California San Francisco Benioff Children's Hospital, Oakland, CA
Mary Eapen, Medical College of Wisconsin, Milwaukee, WI
Yiwen Liu, Dana Farber Cancer Institute, Boston, MA
Fuad El Rassi, Emory University School of Medicine, Atlanta, GA
Edmund K. Waller, Winship Cancer Institute of Emory University, Atlanta, GA
John E. Levine, Icahn School of Medicine at Mount Sinai, NY
John J. Strouse, Duke University, Durham, NC
Joseph H. Antin, Dana Farber Cancer Institute, Boston, MA
Suhag H. Parikh, Emory University School of Medicine, Atlanta, GA
Nitya Bakshi, Yale School of Medicine, New Haven, CT
Carlton D Dampier, Emory University, Atlanta, GA
Jennifer J. Jaroscak, Medical University of South Carolina, Charleston, SC
Shayla Bergmann, Medical University of South Carolina, Charleston, SC
Trish E. Wong, Oregon Health and Science University, Portland, Oregon
Vamsi K. Kota, Georgia Cancer Center at Augusta University, Augusta, GA
Betty Sue Pace, Augusta University, Augusta, GA
Lazaros J. Lekakis, University of Miami, Miami, FL
Premal D. Lulla, Baylor College of Medicine, Houston, TX
Robert Nickel, Children's National Hospital, Washington, District of Columbia
Kimberly A. Kasow, University of North Carolina at Chapel Hill, Chapel Hill, NC
Uday R. Popat, University of Texas M.D. Anderson Cancer Center, Houston, TX
Wally R. Smith, Virginia Commonwealth University, Richmond, VA
Lolie C. Yu, LSU Health Sciences Center - New OrleansFollow
Nancy L. DiFronzo, National Heart, Lung, Blood Institute, Bethesda, MD
Nancy L. Geller, National Heart, Lung, Blood Institute, Bethesda, MD
Naynesh Kamani, Children's National Hospital, Washington, District of Columbia
Elizabeth Sue Klings, Boston University, Boston, MA
Kathryn Hassell, University of Colorado, Aurora, CO
Adam M. Mendizabal, EMMES Corporation, Rockville, MD
et al

Document Type

Article

Publication Date

10-29-2024

Publication Title

Blood Advances

Abstract

Disease-modifying therapies are standard of care (SOC) for sickle cell disease (SCD), but hematopoietic cell transplantation (HCT) has curative potential. We compared outcomes prospectively through 2-years after biologic assignment to a Donor or No Donor (SOC) Arm based on the availability of an HLA-matched sibling or unrelated donor (BMTCTN 1503; NCT02766465). A donor search was commenced after eligibility confirmation. The primary endpoint was the comparison of survival 2 years after biologic assignment between treatment arms. Power calculations required 60 participants on the Donor Arm and 140 on the No Donor Arm to determine if early transplant-related mortality might be balanced by disease-related mortality over a longer period of follow-up. Secondary objectives compared changes in SCD-related events, functional outcomes, and organ function. Data were analyzed by the intent-to-treat principle. A total of 113 participants were enrolled, 28 on the Donor and 85 on the No Donor Arm The 2-year probabilities of survival were 89% and 93%, on the Donor and No Donor Arms, respectively. Vaso-occlusive pain (VOC) was less frequent on the Donor Arm in the second year after biologic assignment (p < 0.001). On PROMIS-57 surveys there was decreased fatigue (p=0.003) and an increased ability to participate in social roles and activities (p=0.003) on the Donor Arm 2-years after biologic assignment. Differences in other secondary outcomes did not reach statistical significance. Barriers to accrual prevented an objective comparison of survival. Assignment to the Donor Arm led to improvements in VOC, fatigue, and social function.

PubMed ID

39471440

Comments

See article for full author list.

Download

Share

COinS