Long-Term Quality of Life Remains Normal After HCT for Sickle Cell

Long-term health-related quality of life (HrQoL) remained largely within population norms after the use of allogeneic hematopoietic cell transplant (HCT) as a curative therapy for sickle cell disease (SCD), according to new findings.¹

This interim analysis of the Sickle Cell Transplant Evaluation of Long-Term and Late Effects Registry (STELLAR) represented the first assessment of prospective HrQoL in long-term survivors of HCT for SCD.

“We describe long-term HrQoL in patients who received HCT for SCD, thus beginning to respond to the mandate of the American Society of Hematology and the Center for Medicare Services to incorporate patient-reported outcomes (PROs) and other nontraditional outcomes in HCT studies,” wrote Lakshmanan Krishnamurti, MD, chief of pediatric hematology, oncology, and bone marrow transplant, Yale School of Medicine.

Nearly 100,000 people in the US, and millions globally, are impacted by SCD, a disease characterized by acute and chronic pain, as well as progressive organ damage.² Although limited, preventive therapies can reduce complications and benefit HrQoL, but rely upon patient treatment adherence, indicating the importance of studying PROs in SCD.

HCT has been shown to prevent or reverse end-organ complications with strong survival, particularly in young children.³ Survival and graft versus host disease (GVHD) rates remain critical measures of treatment success, but PROs can measure the overall impact of HCT.

STELLAR enrolled children and adults ≥ 1 year post-HCT, who completed surveys using a web-based platform.¹

Individuals aged 8–17 completed the Patient Reported Outcome Measurement Information System (PROMIS)-25 version containing 6 HrQoL domains, including mobility, anxiety, depression, fatigue, peer relationships, and pain interference. Those aged ≥18 completed PROMIS-29 evaluating 7 health domains, including physical function, fatigue, pain interference, depression, anxiety, social participation, and sleep disturbance.

Overall, 27 individuals completed PROMIS-25, and 16 completed PROMIS-29 between 2017 and 2021. The median age at HCT was 7.4 and 14.5 years, respectively, with a median age of 10.9 and 22.9 at survey completion.

Upon analysis, the mean PROMIS-25 T-scores for anxiety (43.22), depression (42.94), fatigue (39.25), pain interference (41.70), and peer relationships (48.04) were within the normal range. On the other hand, PROMIS-25 T-scores for mobility experienced a mild decrease below the norm (41.4).

Mean PROMIS-29 T-scores for physical function (52.26), anxiety (53.36), depression (52.54), fatigue (47.70), pain interference (51.71), and sleep disturbance (53.14) additionally remained within the normal range. Social participation T-scores in PROMIS-29 were within the moderate range below the norm (34.3).

Mobility univariate analysis in PROMIS-25 was not significant. Univariate analysis in PROMIS-29 on social participation showed a statistically significant score increase (0.6 [95% CI, 0–1.1; P = .037) with older age at HCT. Physical function univariate analysis in PROMIS-29 revealed a significant reduction with older age at HCT (–0.8 [95% CI, –1.3 to –0.3]; P = .005).

Importantly, men demonstrated statistically significantly lower fatigue than women (–11.9 [95% CI, –23.3 to –0.5]; P = .042).

In their conclusion, Krishnamurti and colleagues indicated the necessity of further accrual and case-control analysis in STELLAR to determine if the findings are sustained and to delineate any factors contributing to PROs.

“Together, these studies have the potential to identify any modifiable variables, including treatment-related factors that could further optimize HrQoL for this SCD population post-HCT,” they wrote.

References

1. _{Arnold SD, Bakshi N, Ross D, et al. Long-term quality of life after hematopoietic cell transplant for sickle cell disease in childhood: A STELLAR interim analysis. Am J Hematol. Published online August 6, 2024. doi:10.1002/ajh.27436}
2. _{Badawy SM, Beg U, Liem RI, Chaudhury S, Thompson AA. A systematic review of quality of life in sickle cell disease and thalassemia after stem cell transplant or gene therapy. Blood Adv. 2021;5(2):570-583. doi:10.1182/bloodadvances.2020002948}
3. _{Stenger EO, Shenoy S, Krishnamurti L. How I treat sickle cell disease with hematopoietic cell transplantation. Blood. 2019;134(25):2249-2260. doi:10.1182/blood.2019000821}