National trends in hydroxyurea and opioid prescribing for sickle cell disease by office‐based physicians in the United States, 1997‐2017

Abstract

Purpose To identify trends in physician drug prescribing practices for sickle cell disease (SCD). Methods We used data from the National Disease and Therapeutic Index to evaluate medications prescribed to children (definition: aged 19 years or younger) and adults (20 years or older) with SCD by office‐based physicians in the United States during 1997 to 2017. Prescriptions were evaluated in 3‐year intervals. Results The proportion of SCD visits that included new/continued hydroxyurea prescriptions increased from less than or equal to 8% before 2009 to 33% in 2015 to 2017. The increase was significant in visits by children (2.5% in 1997‐1999 to 47% in 2015‐2017; P = .003 by Spearman's rank‐order correlation) but not in adults (6.9% to 11%; P = .12). Opioids, started/continued in 13% (lowest 3‐year average) to 35% (highest) of visits by children and 55% to 81% of visits by adults, remained the most frequently prescribed medications for SCD overall. There were no significant changes over time in opioid prescribing for adults (P = .64) or children (P = .38). Hematologists/oncologists accounted for a higher proportion of visits by children (67.2% over 1997‐2017) than adults (25.2%), while emergency medicine visits were higher in adults (14.0%) than children (2.6%). Conclusions This study suggests a robust increase in hydroxyurea prescribing for children with SCD. The BABY HUG trial, which demonstrated safety and efficacy of starting hydroxyurea in infancy and informed current SCD guidelines recommending broader use in children, may have contributed to this increase. However, hydroxyurea prescribing for adults remains infrequent and considerably lower than opioids. Barriers in access to specialist care persist for adults with SCD.

Hydroxyurea Optimization through Precision Study (HOPS): study protocol for a randomized, multicenter trial in children with sickle cell anemia

“…19 While the 2014 guidelines increased the early initiation of hydroxyurea, use remains concerningly low with recent data suggesting that less than 50% of children with SCA are prescribed this life-saving medication. 20,21 In addition to the importance of early initiation of hydroxyurea to prevent SCA complications, our experience documents that hydroxyurea dosing is also a critically important determinant to optimize the clinical and laboratory effects of the medication. The clinical bene ts of hydroxyurea are maximized when the HbF production is optimized and HbF effect is largely determined by degree of hydroxyurea exposure.…”

Association Between Vaso-Occlusive Crises and Opioid Prescriptions Among Patients with Sickle Cell Disease: A Retrospective Claims-Based Study

“…Due to the lack of specialists, adult SCD patients visit the ED more often and visit hematology/oncology specialists less often than do children. 52 When an SCD pain crisis does occur, it should be treated appropriately and adequately. As found in the CDC's clarification letter responding to concerns issued by the American Society of Clinical Oncology, the American Society of Hematology, and the National Comprehensive Cancer Network, special consideration should be given to SCD patients' pain management, and access to appropriate pain management should not be limited.…”

“…33 , 49 , 50 Hydroxyurea has shown its beneficial effects in reducing the number of VOC events for the last two decades. 19 , 24 , 25 , 51 However, hydroxyurea prescribing is infrequent and even more infrequent than opioid prescribing among adults with SCD, 52 which could be due to physicians’ concerns about blood-monitoring requirements and patients’ general nonadherence with the therapy. 53 , 54 L-glutamine oral powder, approved by the US Food and Drug Administration in July 2017, can be added to hydroxyurea to further reduce VOC events.…”

The Evolving Pharmacotherapeutic Landscape for the Treatment of Sickle Cell Disease

“…The BABY HUG trial, which demonstrated safety and efficacy of starting HU in infancy contributed to a robust increase in HU prescribing for children with SCD. 62 Hydroxyurea use in infants 5-12 months old resulted in a better response compared with use in older patients. 63 Moreover, prospective longitudinal follow-up of children with SCD treated with HU since infancy was highly effective in preventing complications of SCD.…”