Patients, Parents and Providers Perspectives Toward Treatment Options for Sickle Cell Disease

Introduction

Sickle cell disease (SCD) is the most common hemoglobinopathies in the world. It is more prevalent in malaria endemic areas where most low-and-middle-income countries (LMIC) are found. Because of challenges in accessing care, persons with SCD (PWSCD) in these settings have a poor outcome and up to 50% die before the age of five years in some places. Those who survive early childhood develop complications at a young age causing their families significant emotional and socioeconomic distress. Effort in LMIC is being directed toward screening for SCD and early initiation of treatment which has improved overall survival and quality of life of PWSCD. However, there are concerns about adherence to treatment due to pill fatigue, persistent SCD symptoms, end organ damage despite available treatment, and side effects of medications. With improved care, SCD is evolving into a chronic illness even in LMIC and PWSCD are surviving into adulthood. These countries will soon face the challenge of handling lifestyle and health demands of an emerging adult and aging PWSCD. There have been significant advances in disease modifying (DM) and curative treatment for SCD in high-income-countries (HIC) and the two promising treatment modalities for cure are bone marrow transplant and gene therapy which can be life changing for PWSCD and their families despite treatment related adverse events. Curing SCD may help prevent long-term complications. It is therefore important to explore the perspectives of PWSCD, their parents and SCD providers toward traditional and novel therapies for SCD as the initial step in planning for improved access to SCD treatment in LMIC.

Objective

To explore the attitudes of PWSCD, Parents (caregivers) of PWSCD and SCD care providers toward traditional and novel therapies for SCD in a LMIC setting.

Methodology

This was a mixed methods study conducted at a referral facility for PWSCD in western Kenya. It included 5 focused group discussions (FGD) of 6 - 7 consented participants; 2 with patients, 2 with parents and 2 with SCD providers. FGDs were conducted by trained clinicians and lasted 2 hours. A discussion guide which included information on novel therapies was applied to direct the audio-recorded sessions conducted in local dialect. FGD and analysis were iterative with each thematic content analyzed independent of the others and key concepts arising from these sessions explored. Necessary adjustments were made to the discussion guide for subsequent sessions based on emerging themes. The recorded material was then transcribed in verbatim and translated for analysis using a qualitative analysis tool.

Results and Discussion

This study enrolled 13 PWSCD, 13 parents of PWSCD and 6 SCD providers (n=32). Participants had similar attitudes towards SCD barriers and novel treatments. 96.9% of participants reported using hydroxyurea and folic acid for themselves, their loved one, or their PWSCD. Over the past year, 83.1% of PWSCD reported painful crisis while 33.% had been hospitalized despite adherence to treatment (table 1). Most participants (65.4%) would consider using novel SCD treatments with hesitations being cost, treatment adverse effect, and distance to treatment location. After the educational intervention, participants were more likely to opt for gene therapy (65.4%) than stem cell transplant (19.2%) or remaining on their current regimen (15.6%) (Table 2). Only one SCD providers' FGD was conducted due to their limited availability from competing clinical duties. Some patients who reported being on recommended treatment continued to have health events and complications that made them, their parents or providers want an alternative treatment. Most participants preferred a curative treatment option for their disease despite the challenges mentioned, which highlighted their willingness to participate in clinical trials. While there are infrastructural challenges in LMIC, they have the highest number of PWSCD that would benefit from new DM and curative treatment for SCD despite being underrepresented in clinical trials. They are the most impacted by complications associated with SCD due to sub-optimal care thus need for action to reverse this.

Conclusion

This study highlights the importance of involving population in LMIC in efforts to find better treatment for PWSCD. There is a need to conduct a similar study involving a larger cohort in future to further explore more perspectives.