Donor health-related quality-of-life and physician decision-making in the context of haploidentical hematopoietic stem cell transplantation (HaploQOL)

Abstract Hematopoietic stem cell transplantation (HSCT) is a preferred treatment for life-threatening diseases and cancers of the blood. HSCT involves exposing the patient to toxic doses of chemotherapy or radiation to destroy diseased cells followed by an infusion of healthy donor cells to rescue the compromised immune system. Worldwide, more than one million HSCT have been performed and ~8,000 HSCT are performed in the US each year. However, challenges in identifying matched sibling or unrelated donors means that a large percentage of patients are unable to find a suitable donor. Recent advances in the prevention of transplant- related complications have allowed for safe and effective transplants using haploidentical (haplo), or half- matched donors. These donor-recipient pairs are most often parent to child or child to parent. Mounting evidence that using haplo donors can produce excellent clinical outcomes has resulted in a 20% increase in haplo transplantation across a recent 4 year period, and haplo transplantation now comprises >25% of all US transplants. However, little is known about the health-related quality-of-life (HRQoL) and donation-related experiences of haplo donors. There is reason to believe that HRQoL in parent-child donation may differ from that of sibling donation due to the nature of the relationship – particularly when the parent/child recipient has compromised health status or does not survive. Additionally, little is known about how physicians make the decision to use a haplo donor versus another source when one is available (e.g., matched unrelated donor), and there is wide variation in the frequency of haplo donation by transplant center. The ultimate goals of this project are to examine HRQoL and factors associated with HRQoL among haploidentical donors and to describe current practice patterns and decision-making about haplo donation among transplant physicians. Specifically, we will (1) examine HRQoL and donation experiences among haplo donors in a series of 6 focus groups with adult and adolescent haplo donors to identify themes in their donation-related experiences and HRQoL, (2) examine HRQoL in a retrospective cohort of adult and adolescent haplo donors who are at one year post-donation using cross-sectional structured telephone interviews with 350 haplo donors (300 adults and 50 adolescents aged 13-17), (3) compare haplo donor HRQoL to that of historical comparison groups of matched unrelated donors and sibling donors, and (4) examine physician decision-making and transplant center selection patterns in the context of haplo donation via 3 physician focus groups and web-based survey with ~170 US-based transplant centers. This study is important because parent-child donation has not been systematically investigated in a nationwide sample and there are reasons to believe that donations between parents and children may make donors particularly vulnerable. The investigation is also novel because there have been no other systematic nationwide investigations of haplo donation. This investigation will provide the foundation for recommendations and guidelines directed toward the selection of and care for haplo donors.