Hydroxyurea decreases hospitalizations in pediatric patients with Hb SC and Hb SB+ thalassemia

Abstract

PurposePatients with hemoglobin SC (Hb SC) and hemoglobin SB+ (Hb SB+) thalassemia suffer from frequent hospitalizations yet strong evidence of a clinical benefit of hydroxyurea (HU) in this population is lacking. Patients with recurrent hospitalizations for pain crisis are offered HU at our institution based on small cohort data and anecdotal benefit. This study identifies outcomes from a large cohort of patients with Hb SC and SB+ thalassemia who were treated with HU for 2 years.Materials and methodsA retrospective review was conducted of 32 patients with Hb SC and SB+ thalassemia who were treated with HU. We reviewed the number, and reasons for hospitalization in the 2 years prior to, and 2 years post-HU treatment as well as laboratory changes from baseline, over 1 year.ResultsPatients with Hb SC and SB+ thalassemia started on HU for frequent pain, had a significant reduction in hospitalizations over 2 years as compared to the 2 years prior to HU initiation (mean total hospitalizations/year: pre-HU: 1.6 vs post-HU 0.4 hospitalizations, P<0.001; mean pain hospitalizations/year: pre-HU 1.5 vs post-HU 0.3 hospitalizations, P<0.001). Patients demonstrated hematologic changes including an increase in percent fetal hemoglobin (%HbF) pre–post HU (4.5% to 7.7%, P=0.002), mean corpuscular volume (74 to 86 fL, P<0,0001), and decrease in absolute neutrophil count (5.0 to 3.2×109/L, P=0.007). Patients with higher doses of HU demonstrated the greatest reduction in hospitalizations but this was unrelated to absolute neutrophil count.ConclusionThis cohort of patients with Hb SC and SB+ thalassemia provides additional support for using HU in patients with recurrent hospitalizations for pain. A large randomized multicenter trial of HU to reduce pain admissions should be conducted to confirm these data and provide much needed evidence based recommendations for this population.

Hydroxyurea prescription, availability and use for children with sickle cell disease in Italy: Results of a National Multicenter survey

“…Second, the SC/Sβ+ group was small and therefore the information on HU use and efficacy in this group of patients is still limited. Since increasing evidence in various settings shows benefit of HU treatment in this subgroup of patients and the number of immigrants from West Africa, where SC disease is common, is increasing rapidly in Italy, the current limitation will be overcome in the future. Third, although the Italian healthcare system for rare hematological disorders provides that children with SCD receive care in specialized pediatric hematology centers, children living in rural areas or immigrant children recently arrived in Italy might be taken in care in local hospitals and therefore the number of overall patients and of those in HU therapy is probably underestimated; the lack of a national newborn screening program with standardized data collection and the absence of a national hemoglobinopathy registry could easily overcome this issue and are currently under development.…”

Barriers to hydroxyurea use from the perspectives of providers, individuals with sickle cell disease, and families: Report from a U.S. regional collaborative

“…Recurrent and repetitive VOEs may lead to frequent healthcare visits or interference with daily functioning, even in the absence of healthcare utilization, that negatively affect the quality of life for children and adults with SCD [National Heart, Lung and Blood Institute, National Institutes of Health (NHLBI, NIH), 2014; Jerrell et al, 2011;Tanabe et al, 2012]. Long-term management of SCD requires chronic medical care with access to education about prevention as well as current and future disease modifying and curative therapies (Wang et al, 2011;Alvarez et al, 2013;Yates et al, 2013;DeBaun et al, 2014;Lebensburger et al, 2015;McGann and Ware, 2015;Fitzhugh et al, 2017;Niihara et al, 2018;Quinn, 2018;Krishnamurti et al, 2019). In addition to hydroxyurea, other recently FDA approved therapies include voxelotor, L-glutamine, and crizanlizumab (Ataga et al, 2017;Howard et al, 2019;Vichinsky et al, 2019).…”

Stroke in a Child with Hemoglobin SC Disease: A Case Report Describing Use of Hydroxyurea after Transfusion Therapy

“…Given the challenges of chronic red cell transfusions in this population, alternative therapy is needed. Recent single institution retrospective studies involving HbSC patients 25 suggest HU reduces pain crises making it a feasible option. Data from a multicenter retrospective study of several HbSC cohorts support the ability of HU to increase HbF and decrease white cell counts without severe bone marrow suppression in adults and children to produce a beneficial effect.…”