Strategies to increase access to basic sickle cell disease care in low- and middle-income countries

ABSTRACT

Introduction

Sickle cell disease (SCD) is the most common hemoglobinopathy in the world. Over 90% of those born with SCD live in low- and middle-income countries (LMICs), yet individuals in these settings have much poorer outcomes compared to those in high-income countries.

Areas Covered

This manuscript provides an in-depth review of the cornerstones of basic SCD care, the barriers to implementing these in LMICs, and strategies to increase access in these regions. Publications in English language, peer-reviewed, and edited from 2000 to 2021 were identified on PubMed. Google search was used for gray literature.

Expert Opinion

Outcomes for patients with SCD in high-income countries have improved over the last few decades due to the implementation of universal newborn screening programs and use of routine antimicrobial prophylaxis, increase in therapeutic and curative options, and the adoption of specific measures to decrease risk of stroke. This success has not translated to LMICs due to several reasons including resource constraints. A combination of several strategies is needed to increase access to basic SCD care for patients in these settings.

KEYWORDS:

• Global health
• sickle cell disease
• low- and middle-income countries
• newborn screening
• penicillin
• hydroxyurea
• stroke
• voxelotor
• crizanlizumab
• l-glutamine

Article Highlights

• Over 300,000 babies are born with SCD annually. Approximately 90% of these births take place in LMICs.

• The highest incidence of disease is seen in SSA, where 50–90% of children with SCD die before they reach age five.

• Effective management for SCD begins with early diagnosis via NBS. However, the areas with the highest burden of SCD often lack universal NBS programs.

• SCD is associated with increased susceptibility to infection secondary to encapsulated bacteria. Current recommendations include the use of prophylactic antimicrobials and the completion of the pneumococcal vaccine series. However, the availability of immunizations and penicillin is limited in LMICs.

• Hydroxyurea decreases the frequency of acute and chronic complications of SCD and is standard of care for patients with SCD in high-income countries. The majority of patients in LMICs still do not have access to this life-saving medication.

• Children with SCD are at increased risk of ischemic stroke. In high-income countries, EBP for primary stroke prevention includes screening for abnormal transcranial Doppler ultrasound velocity and use of blood transfusion therapy for repeated abnormal velocities. Unfortunately, this can often not be implemented in low-resource settings.

• HLA-matched sibling donor hematopoietic stem cell transplantation is standard of care curative therapy for SCD. However, this is an expensive and resource-intensive procedure that is not accessible for most patients in LMICs.

• Many strategies are being used to increase access to care for SCD patients in LMICs. Strategies include establishing public–private partnerships, training CHW to support SCD management, engaging local government officials and religious leaders to develop culturally competent programs, and developing locally relevant educational materials for patients and families.

Declarations of Interest

JH Estepp receives consultant fees from Daiichi Sankyo, Global Blood Therapeutics, Emmanus Life Sciences, and Agios Pharmaceuticals and research support from Global Blood Therapeutics, Forma Therapeutics, Pfizer, Eli Lily and Company, the American Society of Hematology Scholar Program, and the NIH.

H Bello-Manga is supported by the NIH (K43TW011583) and is an investigator for a Global Blood Therapeutics trial (Hope Kids2).

YM Carroll receives consultant fees from Forma Therapeutics and Global Blood Therapeutics.

AA King is supported by the NIH (1K24HL148305, 5U01HL133994) and HRSA (6U1EMC27865). AA King also receives research funding from Global Blood Therapeutics.

The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer Disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.