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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.

Summary Objective Cleft lip and/or palate (CLP) is the most common congenital craniofacial anomaly. As a first step toward developing a quality of life (QOL) questionnaire for CLP patients, our team conducted a systematic literature review to identify studies that measured child- or proxy-reported outcomes of CLP. Design PUBMED, CINAHL, EMBASE and PsycINFO were searched from their inception to July 2010 to identify studies that measured health-related concepts in CLP patients. Abstract and title screening was performed by two screeners. Full texts of all potentially relevant papers were obtained and examined by two reviewers. We identified publications that measured health concepts and categorized them to form a preliminary conceptual framework of CLP QOL issues. Results A total of 4594 publications were identified. Twenty-six studies met our inclusion criteria. Research involved CLP patients living in nine countries with sample sizes ranging from 23 to 661. Health concepts were measured using 29 different questionnaires. No patient-reported outcome (PRO) instrument measuring QOL concerns of CLP patients currently exists. CLP-specific health concepts measured to date were categorized into a preliminary conceptual QOL framework with the following categories: physical, psychological and social health. Conclusions Our review has helped to identify areas of health that have been well researched using either a patient or proxy patient-reported outcome instrument (e.g., self-concept; behavior) and areas where more research is required.