Session: 901. Health Services Research—Non-Malignant Conditions: Poster II
Hematology Disease Topics & Pathways:
sickle cell disease, Diseases, Hemoglobinopathies, Clinically relevant
Methods: DISPLACE data collection years were 2012 – 2016. 5247 children with SCA who met criteria for annual TCD screening were entered into the electronic data capture system with clinical and demographic information. 163 children with abnormal TCD results were identified from the cohort database. Additional data were collected and subsequently adjudicated including timing of follow-up testing (repeat TCD or MRI) after abnormal TCD, implementation of CRCT following abnormal TCD, alternate treatment following abnormal TCD, and whether the STOP protocol was implemented. Data were analyzed using descriptive statistics.
Results: In response to abnormal TCD, 75% of children had a repeat TCD ordered (Table 1). Of these, most repeat TCDs were completed within 3 months, with <10% delayed (longer than 12 weeks). CRCT was initiated in 69% of children with abnormal TCD (Table 2); of these, 17% had delayed initiation of CRCT. After abnormal TCD, 10% of families declined CRCT though suggested by providers. When some of these children later suffered an ischemic stroke, (20%) started CRCT at that time. The TWiTCH protocol (CRCT transitioning to HU in children without severe vasculopathy) was followed in about 1/4 of children on CRCT. Additional treatments following abnormal TCD included initiation of HU (64%) and bone marrow transplant (4%) (Table 3). Characteristics of children on CRCT are described in Table 4. Notably, of the 19 children whose CRCT was delayed, 15 (79%) had a stroke. There were 48 instances in which the STOP protocol was not implemented following abnormal TCD. Half of these were the result of decisions at the parent level (e.g., the parent refused CRCT, parent requested HU instead of CRCT). Five were provider decisions (e.g., provider suggested HU), and 15 were systems-level issues (e.g., repeat TCD was delayed). One was a combination of parent decision and system-level issue, and in 4 cases, the reason was unknown.
Conclusions: In this nationally representative sample of children with SCA at risk for stroke, nearly ¾ of children had a repeat TCDs, suggesting confirmation of abnormal results was common prior to beginning CRCT. Notably, there were a large number of ischemic strokes in children when CRCT was delayed futher confirming the utility of the screening and stroke prevention protocol. An additional key concern identified in this study were the barriers to STOP protocol implementation. Common reasons for lack of implementation included the parent’s decision for HU instead of CRCT, delays in CRCT initiation, and delays in obtaining repeat TCDs. Difficulties implementing the STOP protocol suggest the need for interventions for more rapid initiation of CRCT and stroke prevention.
Disclosures: Kanter: Novartis: Consultancy; AGIOS: Membership on an entity's Board of Directors or advisory committees; SCDAA Medical and Research Advisory Board: Membership on an entity's Board of Directors or advisory committees; BEAM: Membership on an entity's Board of Directors or advisory committees; NHLBI Sickle Cell Advisory Board: Membership on an entity's Board of Directors or advisory committees; Wells Fargo: Honoraria; Cowen: Honoraria; Jeffries: Honoraria; GLG: Honoraria; Guidepoint Global: Honoraria; Sanofi: Consultancy; Medscape: Honoraria; bluebird bio, inc: Consultancy, Honoraria.
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