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abstracts & posters

Pediatric prescriber pratices of hyrdoxyurea in sickle cell disease

key information

source: American Society of Pediatric Hematology/Oncology

year: 2016

authors: Anjulika Chawla, Philippa Sprinz, Patricia Kavanagh


Hydroxyurea (HU) has been shown to be efficacious in individuals with sickle cell disease (SCD). Drug labeling gives prescribing guidance for use in adults, but use in children is based on expert consensus and not randomized trials. This leads to variation in HU initiation, counseling, monitoring, and discontinuation.

Design/Method: We conducted an online survey of HU prescribing practices among providers in the New England Pediatric Sickle Cell Consortium caring for approximately 1000 children with SCD, from September-October 2013.

Results: We had responses from 13 providers from 10 New England institutions. Six providers estimated that they had > 50% of eligible patients on HU. HU initiation indications used by >70% of respondents included: all patients with hemoglobin SS and SB0-thalassemia, conditional TCD velocities, >=3 pain episodes/year, multiple hospitalizations, and parental request. All providers felt HU was safe for children >2 years old, 7/11 offered it in children <2 years. Respondents reported that patient or family declining was the most common reason for not initiating HU; reasons given included the increased frequency of office visits, difficulty of daily medications, fear of side effects, concerns for dependency, and loss of fertility. Other reasons for ineligibility included hepatic or renal dysfunction, splenic sequestration, neutropenia, high baseline hemoglobin, and history of poor adherence. 90% of prescribers either provided or referred patients for counseling about birth control , 7/12 performed urine pregnancy tests routinely, and 33% of providers required documentation of birth control in females. Respondents typically started HU at 15-20 mg/kg/d, but maintenance doses varied widely, from 15 to 35mg/kg/d. Lab monitoring was done every 2-4 weeks during escalation, and every 2-3 months with stable dosing. Adherence was most commonly monitored by self-report and inferred by lab values. The top three indications to discontinue HU included missed visits, poor adherence or neutropenia.

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