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Summary: Health‐related quality of life (HRQoL) is an important outcome for patients with sickle cell disease (SCD). It is often poor compared with other chronic medical conditions or measured as a multidomain disease‐specific construct. We previously reported outcomes in the Start Healing in Patients with Hydroxyurea (SHIP‐HU) randomized controlled trial in adolescents and adults with SCD at six clinical sites. Besides the primary outcomes, we also measured HRQoL as a secondary outcome. Patients in the intervention arm were each assigned community health workers (CHWs) who provided case management services. CHW services were independent of medical management, and medical managers were blinded to the study arm. Patients in the control arm received only standard of care. We hypothesized that having a CHW would improve HRQoL in patients enrolled in SHIP‐HU. We did not find significant differences between domains of HRQoL in the two study arms. Possible explanations include selection bias of enrolled versus unenrolled patients, selection bias of sites, medical providers and medical management, enforced blinding, and a lack of cooperation between medical managers and CHWs. The importance of CHWs and HRQoL is nonetheless recognized based on the literature. Future interventions on HRQoL in SCD should consider alternative study designs and multimodal interventions. [ABSTRACT FROM AUTHOR]

Summary: Hydroxyurea (hydroxycarbamide) (HU) for sickle cell anaemia (SCA) is underutilised. Case management is an evidence‐based health management strategy and in this regard patient navigators (PNs) may provide case management for SCA. We hypothesised that HU‐eligible patients exposed to PNs would have improved indicators of starting HU and HU adherence. We randomised 224 HU‐eligible SCA adults into the Start Healing in Patients with Hydroxyurea (SHIP‐HU) Trial. All patients received care from trained physicians using standardised HU prescribing protocols. Patients in the Experimental arm received case management and education from PNs through multiple contacts. All other patients were regarded as the Control arm and received specialty care alone. Study physicians were blinded to the study arms and did not interact with PNs. At baseline, 6 and 12 months we assessed and compared laboratory parameters and HU adherence indicators. Experimental patients had higher 6‐month mean fetal haemoglobin (HbF) levels than controls. But at 12 months, mean HbF was similar, as were white blood cell count, absolute neutrophil count, total haemoglobin, platelet count and mean corpuscular volume. At 12 months there were fewer experimental patients missing HU doses than controls (mean 1·8 vs. 4·5, P = 0·0098), and more recent HU prescriptions filled than for controls (mean 53·8 vs. 92 days, median 27·5 vs. 62 days, P = 0·0082). Mean HU doses were largely similar. We detected behavioural improvements in HU adherence but no haematological improvements by adding PNs to specialty care. [ABSTRACT FROM AUTHOR]

We sought to investigate whether hydroxyurea therapy is associated with the need for surgical splenectomy among patients with sickle cell disease (SCD). We hypothesized that as hydroxyurea gained widespread use, surgical splenectomy among pediatric patients with SCD occurred at a higher rate and older age among those taking hydroxyurea. In this retrospective cross-sectional study, the Pediatric Health Information System was queried for all SCD International Classification of Diseases 9/10 diagnosis codes and splenectomy procedure codes from January 1, 2005, to December 31, 2020. Hydroxyurea use was defined as at least one hospital admission with hydroxyurea listed as a medication. The rates of surgical splenectomy, age at splenectomy, hospital length of stay, and incidence of blood transfusion during the splenectomy admission were compared among patients receiving hydroxyurea versus those not receiving hydroxyurea. Additional subanalysis was performed in the Hemoglobin-SS, Hemoglobin-SC, and Other cohorts separately. During the study period, 28,520 patients were identified. All patients with SCD receiving hydroxyurea had a significantly higher rate of surgical splenectomy compared with the nontreatment group (7.2% versus 3.2%, P = 0.01). The age at surgical splenectomy was significantly younger among Hemoglobin-SS patients receiving hydroxyurea (5.7 [5.1, 6.4] y versus 6.6 [5.8, 7.4] y; P < 0.01). There were no significant differences in length of stay or incidence of blood transfusion during the surgical splenectomy admission between treatment groups. Hydroxyurea use in children is associated with higher rates of surgical splenectomy and occurs at a younger age in the Hemoglobin-SS population. Although these findings warrant further investigation for causality, it provides useful information to clinicians and patients alike, allowing for more informed decision-making. [ABSTRACT FROM AUTHOR]