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Master of Public Health

Long-term organ function after HCT for SCD: a report from the Sickle cell Transplant Advocacy and Research alliance

AUTHORS

Stenger E , Xiang Y , Wetzel M , Gillespie S , Chellapandian D , Shah R , Arnold SD , Bhatia M , Chaudhury S , Eckrich MJ , Kanter J , Kasow KA , Krajewski J , Nickel RS , Ngwube AI , Olson TS , Rangarajan HG , Wobma H , Guilcher GM , Horan JT , Krishnamurti L , Shenoy S , Abraham A , . Transplantation and cellular therapy. 2022 10 20; ().

ABSTRACT

Hematopoietic cell transplantation (HCT) is an established cure for sickle cell disease (SCD) supported by long-term survival, but long-term organ function data are lacking. We sought to describe organ function and assess predictors for dysfunction in a retrospective cohort (n=247) through the Sickle cell Transplant Advocacy and Research alliance. Patients with <1 year follow up or graft rejection/second HCT were excluded. Organ function data was collected from last follow up. Primary measures were organ function, comparing pre- and post-HCT. Bivariable and multivariable analyses were performed for predictors of dysfunction. Median age at HCT was 9.4 years; majority had HbSS (88.2%) and severe clinical phenotype (65.4%). Most received matched related (76.9%) bone marrow (83.3%) with myeloablative conditioning (MAC; 57.1%). Acute and chronic graft-versus-host disease (GVHD) developed in 24.0% and 24.8%. Thirteen patients (5.3%) died ≥1-year post-HCT, primarily from GVHD or infection. More post-HCT patients had low ejection or shortening fractions than pre-HCT (0.6%→6.0%, p=.007 and 0%→4.6%, p=.003). The proportion with lung disease remained stable. Eight patients (3.2%) had overt stroke; most had normal (28.3%) or stable (50.3%) brain MRI. On multivariable analysis, cardiac dysfunction was associated with MAC (OR=2.71, 95%CI: 1.09-6.77, p=.033) and severe acute GVHD (OR=2.41, 95%CI: 1.04-5.62, p=.041). Neurologic events were associated with CNS indication (OR=2.88, 95%CI: 2.00-4.12, p<.001). Overall organ dysfunction was associated with age ≥16 years (OR= 2.26, 95%CI: 1.35-3.78, p=.002) and clinically severe disease (OR= 1.64, 95%CI: 1.02-2.63, p=.043). In conclusion, our results support consideration of HCT at younger age and use of less intense conditioning.

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