Quality of Life and Personal Life Goals of Adult Sickle Cell Disease Patients after Curative Allogeneic Stem Cell Transplantation: A Mixed Methods Study

Introduction: Sickle cell disease (SCD) is associated with poor quality of life (QoL) and early mortality. In the Netherlands, allogeneic hematopoietic stem cell transplantation (HSCT) as curative treatment option for adult SCD patients was first introduced in 2018. A thorough understanding of the impact of undergoing HSCT on QoL is essential for health care professionals to be able to meet the needs of this patient population.

Aim: To explore the perspectives of adult SCD patients on the impact of curative HSCT on physical, mental, and social health.

Methods: Adult SCD patients who underwent HSCT at the Amsterdam UMC ≥ 1 year ago were eligible for this study. A mixed methods approach consisting of an exploratory qualitative design and a questionnaire component was used. All patients participated in a semi-structured interview on their experienced QoL and personal life goals. Interviews were audio recorded, transcribed, and coded using MAXQDA® software. Results were analyzed using Thematic Analysis. Additionally, participants completed a Patient Reported Outcome Measurement Information System (PROMIS®) questionnaire measuring 9 item banks on physical, mental, and social health at the time of the interview. Results of the PROMIS® questionnaires (T-scores) were related to reference T-scores of the general Dutch population using descriptive statistics (IBM SPSS statistics 28.0).

Results: Ten SCD patients with a median age of 29.5 years (range 19-49) participated in the study between October 2021 and May 2022. The median time since HSCT was 2.7 years ranging from 1 to 3.5 years. Mean T-scores of all participants fell within normal limits for all 9 PROMIS® item banks, as compared to the Dutch general population (Table 1). However, T-scores varied between patients. Two patients with serious avascular bone necrosis (AVN), developed prior to HSCT, scored mild, moderate, or severe on ≥7 of 9 item banks. Another patient scored mild or moderate on all 3 mental health item banks. These differences between patients, as well as a general satisfaction with their current QoL despite having experienced physical and psychosocial difficulties were reflected in the outcome of the interviews. Eight central themes emerged from the Thematic Analysis: (1) pain, (2) physical wellbeing, (3) mental wellbeing, (4) perspective, (5) counseling/guidance, (6) education and work, (7) family and friends, and (8) activities and participation. These themes were categorized in mental, physical, and social health, following the PROMIS framework, and subsequently described based on four phases: before HSCT, HSCT - 1^st year post transplantation, current situation, and future expectations. Most patients reported to experience improved physical, mental, and social health after HSCT and being able to set and achieve personal life goals. However, many participants also described an (ongoing) mental impact of having had SCD their whole life even after curation by HSCT. They expressed a clear need for psychological counseling before, during, and after HSCT. Furthermore, persistence of pain due to AVN had a negative influence on the perceived physical health of some patients. Several patients experienced a discrepancy between their expectations of HSCT and the outcome; either above- or below-expected.

Conclusion: QoL of adult SCD patients who underwent curative HSCT seemed generally comparable to the Dutch general population; patients reported to experience improved QoL, and the ability to pursue personal life goals. However, the physical, mental, and social impact after curation can be extensive, ranging from positive to negative and differed between patients. Patients expressed a need for psychological support before, during, and after HSCT. Moreover, irreversible SCD-related organ damage can negatively influence the QoL of transplant recipients and patient expectations should be actively discussed by health care professionals before HSCT.

Biemond:Novo Nordisk: Membership on an entity's Board of Directors or advisory committees; CSL Behring: Membership on an entity's Board of Directors or advisory committees; Modus Therapeutics: Membership on an entity's Board of Directors or advisory committees; Chiesi: Membership on an entity's Board of Directors or advisory committees; Celgene: Membership on an entity's Board of Directors or advisory committees; Bluebird Bio: Membership on an entity's Board of Directors or advisory committees; GBT: Research Funding; BMS: Research Funding; BMS: Research Funding; Celgene: Membership on an entity's Board of Directors or advisory committees; Novo Nordisk: Membership on an entity's Board of Directors or advisory committees; CSL Behring: Membership on an entity's Board of Directors or advisory committees; Chiesi: Membership on an entity's Board of Directors or advisory committees; Bluebird Bio: Membership on an entity's Board of Directors or advisory committees; GBT: Membership on an entity's Board of Directors or advisory committees, Research Funding; Sanquin: Research Funding; Novartis: Membership on an entity's Board of Directors or advisory committees, Research Funding. Nur:Novartis: Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau.