Real-World Patient-Reported Treatment Patterns and Treatment Satisfaction for Sickle Cell Disease Patients in the United States
Authors: Colavecchia Carmine , Barcelos Giovanna Tedesco , Wirz Reto , Hines Dionne M. , Purdie David M. , Baker Christine L. , Libby Susanna , Lai Sophie , Meier Emily Riehm , Iqbal Halima , Chatterton Emma , Mellor Jennifer 
Published: December 2024

Introduction: Patients are greatly impacted by the unmet needs associated with treatment of sickle cell disease (SCD). Until recently, few medications were approved for SCD. While published literature focuses on adherence, information is limited about patient treatment satisfaction, patient knowledge of SCD or treatment options, and perceptions of providers' knowledge of SCD. With the approval and the continued development of new treatments for SCD, it is critical to understand the patient perspective. We describe SCD treatment patterns and patient perspectives about SCD treatments, their satisfaction, and knowledge of these treatment options.
Methods:We conducted a cross-sectional study to analyze Adelphi Real World (ARW) survey data collected in the United States between June and July 2024. ARW partnered with a patient advocacy group to recruit adults living with SCD. Consenting survey respondents completed questionnaires about their demographics and current treatment, including adherence, satisfaction, and knowledge of treatment options. Responses to survey questions were required to complete the questionnaires to limit missing data. We used descriptive statistics to characterize their perspectives.
Results: Of the 96 respondents, the mean (standard deviation) age was 37.1 (10.6) years, 94% were Black, 86% were female. Overall, 95% (n=91) reported receiving medication, supplements, or chronic transfusions for treatment of SCD or SCD symptoms. Respondents reported taking the following treatments for SCD: pain relief (88%, n=84: acetaminophen, cannabis product, topical medications, NSAIDs, opioids, other pain relievers); supplements (86%, n=83: folic acid, zinc, vitamin D); SCD-disease modifying medications (64%, n=61: hydroxyurea [n=48], crizanlizumab [n=11], L-glutamine [n=11]), voxelotor [n=10], and chronic blood transfusions (22%, n=21).
Of the 96 respondents, 91 (95%) self-reported adherence to their current treatments. Overall, approximately two-thirds (63%, n=57/91) of participants reported being 'completely adherent,' that is, taking medicine(s) exactly as recommended by their doctors. Complete adherence by treatment type, was 66% (n=40/61) for SCD-specific treatments; 63% (n=53/84) for pain relief treatment and 61% (n=51/83) for supplements. Of the participants who did not completely adhere (37%, n=34/91), their top 3 reported reasons were 'forgetting to take the medication' (50%, n=17/34), ‘dislike of medication side effects’ (35%, n=12/34), and ‘saving the medication for when I really needed it, to make it last longer‘(32% overall [n=11/34]; 29% [n=9/31] for those taking pain relief medication).
Most respondents (77%, n=70/91) who reported treatment adherence indicated that they were ‘not very happy’ with their current treatment for SCD, specifically SCD-disease modifying medications (79%, n=48/61), pain relief (79%, n=66/84), and supplements (80%, n=66/83). The top reported reasons for not being completely happy with current treatments were feeling tired (47%, n=33), experiencing side effects from the treatments (41%, n=29), and no longer feeling like the treatment was working (39%, n=27).
Nearly half (47%, n=45/96) of all the patient respondents considered themselves as ‘a little bit knowledgeable’ or ‘not at all knowledgeable’ about the available SCD treatment options available for SCD. While most respondents (79%) rated SCD specialists (eg, hematologists) as ‘very knowledgeable’ about SCD, they rated 80% and 63% of emergency/urgent care providers and primary care physicians, respectively, to only be ‘somewhat knowledgeable’ or ‘not at all knowledgeable’ about SCD.
Conclusions: The findingsof this survey highlight that most patients felt dissatisfaction with SCD treatment, primarily due to ‘feeling tired.‘ Although approximately two-thirds of respondents reported using SCD-specific medications, approximately one-third reported incomplete adherence. Undesirable side effects were a main reason for lower self-reported adherence and reduced satisfaction. Almost half of the participants expressed limited knowledge of treatments and more than half perceived that key physicians were not fully informed about SCD, highlighting a knowledge gap. Broadening patient and physician knowledge about SCD, as well as increasing treatment options which offer fewer side effects, may improve patient satisfaction and adherence.