MANAGEMENT OF SICKLE CELL DISEASE: A CLINICAL PRACTICE SURVEY

ABSTRACT

Background: Therapeutic options for Sickle Cell Disease (SCD) have increased recently as well as the development of updated national guidelines. It is not known how these options are being offered or to what degree guidelines are incorporated into clinical practice. Objectives: To assess the clinical practice patterns of providers treating children with SCD. Design/Method: A survey study was performed which included nine sections clinic structure, prophylaxis, immunizations, hydroxyurea, splenic sequestration, stroke, novel therapies, potential curative therapies, and transition. Survey was disseminated over a three-month period via SurveyMonkey, to members of the American Society of Pediatric Hematology-Oncology Hemoglobinopathy Special Interest Group. Results: There were 86 respondents;most were attending/faculty (85%, 73/86) who were part of a university/academic practice (65%, 56/86). Program size was most commonly 50-250 patients (44%, 37/86). Accessibility to support staff in clinic included 95% (81/86) social work;76% (65/86) child life;68% (58/86) nurse coordinator and 34% (29/86) school liaison and 15% (13/86) transition navigator. For preventive care, 72% prescribe penicillin prophylaxis before 2 months of age recommending 100% (83) for HbSS and Sβnull, 72% (60/83) for HbSC and 70% (58/83) for HbSβplus. Influenza was the most common vaccine offered in clinic at 96% (76/79) with 91% (72/79) offering pneumococcal vaccines, 84% (67/79) offering meningococcal vaccines and 50% (40/79) offering COVID vaccines. Transcranial doppler screening was offered in 95% (69/73) but only 42% (31/73) performed MRI screening for silent stroke. Transfusion therapy was recommended for primary stroke prevention by 90% (65/72) and 84% (59/70) attempt to transition to hydroxyurea following TWITCH guidelines. For secondary stroke prevention, 88% (63/72) recommend chronic transfusion therapy. Regarding disease-modifying therapy, 90% (70/78) report starting hydroxyurea routinely in patients with HbSS and Sβnull;initiated at 9 months of age by 69% (54/78). Laboratory monitoring recommended every 3 months for stable dosing by 62% (49/78) and hydroxyurea held by 56% (44/78) if platelets <75,000, 73% (56/78) for neutrophils <1000. New therapies were recommended for patients on hydroxyurea who were still experiencing SCD complications L-glutamine 68% (37/54;crizanlizumab 93% (54/58). Voxelotor was recommended for patients on hydroxyurea with low hemoglobin 65% (43/66). Matched sibling transplant was considered for any disease severity by 55% (38/69). Gene therapy trial is offered on-site by 29% (20/69). Transition programs were endorsed by 61% (42/69), but only 45% (31/68) had dedicated staff. Conclusion: This survey is the only assessment of the application of SCD guidelines in clinical practice.