Research graph
References from Paediatric Drug Optimization for Sickle Cell Disease: priorities for research and development in children. Local targets link to admitted publications; unresolved targets remain external evidence.
Deconstructing sickle cell disease: reappraisal of the role of hemolysis in the development of clinical subphenotypes
10.1016/j.blre.2006.07.001 · 2007 · External reference
Sickle-cell disease
10.1016/s0140-6736(10)61029-x · 2010 · External reference
Pathological basis of symptoms and crises in sickle cell disorder: implications for counseling and psychotherapy
10.4081/hr.2010.e2 · 2010 · External reference
Global, regional, and national prevalence and mortality burden of sickle cell disease, 2000–2021: a systematic analysis from the Global Burden of Disease Study 2021
10.1016/s2352-3026(23)00118-7 · 2023 · External reference
Defining global strategies to improve outcomes in sickle cell disease: a Lancet Haematology Commission
10.1016/s2352-3026(23)00096-0 · 2023 · External reference
Sickle cell disease in sub-Saharan Africa
10.1016/j.hoc.2015.11.005 · 2016 · External reference
Prevalence estimates of sickle cell disease among children and adolescents in sub-Saharan Africa: a systematic review and modelling analysis
10.1016/s2352-4642(26)00048-9 · 2026 · External reference
Evolution of sickle cell disease from a life-threatening disease of children to a chronic disease of adults: the last 40 years
10.1002/ajh.24235 · 2016 · External reference
Sickle cell disease
10.1016/s0140-6736(17)30193-9 · 2017 · External reference
Using disease-modifying therapies in sickle cell disease
10.1182/hematology.2023000485 · 2023 · External reference
Safety of hydroxyurea in children with sickle cell anemia: results of the HUG–KIDS study, a phase I/II trial
1999 · External reference
Hydroxycarbamide in very young children with sickle-cell anaemia: a multicentre, randomised, controlled trial (BABY HUG)
10.1016/s0140-6736(11)60355-3 · 2011 · External reference
Hydroxyurea (hydroxycarbamide) for sickle cell disease
2022 · External reference
The effect of hydroxcarbamide therapy on survival of children with sickle cell disease
10.1111/bjh.12323 · 2013 · External reference
Barriers and facilitators of availability of hydroxyurea for sickle cell disease in Tanzania; a qualitative study of pharmaceutical manufacturers, importers, and regulators
2022 · External reference
Barriers to therapeutic use of hydroxyurea for sickle cell disease in Nigeria: a cross-sectional survey
10.3389/fgene.2021.765958 · 2022 · External reference
A review on disease modifying pharmacologic therapies for sickle cell disease
10.1007/s00277-025-06216-1 · 2025 · External reference
Unresolved reference
2026 · External reference
Unresolved reference
2026 · External reference
Unresolved reference
2021 · External reference
Unresolved reference
2026 · External reference
A phase 3 trial of l-glutamine in sickle cell disease
10.1056/nejmoa1715971 · 2018 · External reference
Unresolved reference
2019 · External reference
Real-world data on efficacy of L-glutamine in preventing sickle cell disease-related complications in pediatric and adult patients
2022 · External reference
Crizanlizumab for the prevention of pain crises in sickle cell disease
10.1056/nejmoa1611770 · 2017 · External reference
Expert consensus on the management of infusion-related reactions (IRRs) in patients with sickle cell disease (SCD) receiving crizanlizumab: a RAND/UCLA modified Delphi panel
10.1007/s00277-024-05736-6 · 2024 · External reference
Crizanlizumab with or without hydroxyurea in patients with sickle cell disease (STAND): primary analyses from a placebo-controlled, randomised, double-blind, phase 3 trial
10.1016/s2352-3026(24)00384-3 · 2025 · External reference
Real-world data of crizanlizumab in sickle cell disease: a single-centre analysis
10.14740/jh1127 · 2023 · External reference
Sickle cell disease
10.1016/s0140-6736(25)02278-0 · 2026 · External reference
Hope on the horizon: new and future therapies for sickle cell disease
10.3390/jcm12175692 · 2023 · External reference
Safety and efficacy of mitapivat in sickle cell disease (RISE UP): results from the phase 2 portion of a global, double-blind, randomised, placebo-controlled trial
10.1016/s2352-3026(24)00319-3 · 2025 · External reference
Multicenter, phase 1 study of etavopivat (FT-4202) treatment for up to 12 weeks in patients with sickle cell disease
10.1182/bloodadvances.2023012467 · 2024 · External reference
Etavopivat in adolescents with sickle cell disease: emerging safety and efficacy findings from the first cohort of the ongoing phase 1/2 hibiscus kids study
10.1182/blood-2025-618 · 2025 · External reference
Tebapivat improves red cell deformability and decreases sickling in patients with sickle cell disease: a phase 1 trial substudy
10.1111/ejh.70048 · 2026 · External reference
Results from a phase 1 study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of tebapivat (AG-946) in patients with sickle cell disease
2024 · External reference
Oral tetrahydrouridine and decitabine for non-cytotoxic epigenetic gene regulation in sickle cell disease: a randomized phase 1 study
10.1371/journal.pmed.1002382 · 2017 · External reference
One cell at a time: HbF distribution in sickle cell disease
10.1182/bloodadvances.2025018293 · 2026 · External reference
Consensus-driven target product profiles for curative sickle cell disease gene therapies
10.1016/j.omtm.2024.101287 · 2024 · External reference
Towards access for all: 1st working group report for the Global Gene Therapy Initiative (GGTI)
10.1038/s41434-021-00284-4 · 2023 · External reference
Africa must participate in finding a gene therapy cure for sickle-cell disease
10.1038/s41591-022-02033-5 · 2022 · External reference
The translational gap for gene therapies in low- and middle-income countries
10.1126/scitranslmed.adn1902 · 2024 · External reference
Access to gene therapy for sickle cell disease in Africa
10.1016/j.ebiom.2025.105713 · 2025 · External reference
Safety of in vivo gene therapy in children: mechanisms and management of liver injury
10.1016/s2352-4642(25)00380-3 · 2026 · External reference
The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective
10.1182/blood.2021010864 · 2021 · External reference
Make gene therapies more available by manufacturing them in lower-income nations
10.1038/d41586-024-02310-y · 2024 · External reference
Scientific advancements in gene therapies: opportunities for global regulatory convergence
10.3390/biomedicines13030758 · 2025 · External reference
Hospital variations in time-to-crisis-resolution among children and adolescents with sickle cell disease
10.1002/ajh.70129 · 2026 · External reference
The challenge of clinical end points in sickle cell disease
10.1182/blood.2023021220 · 2023 · External reference
Patient-reported outcomes in children with sickle cell disease at presentation for an acute pain episode
10.1182/bloodadvances.2021006794 · 2023 · External reference
What is the future of patient-reported outcomes in sickle-cell disease?
10.1080/17474086.2020.1830370 · 2020 · External reference
End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain
10.1182/bloodadvances.2019000882 · 2019 · External reference
A multicenter randomized controlled trial of intravenous magnesium for sickle cell pain crisis in children
10.1182/blood-2015-05-647107 · 2015 · External reference
Arginine therapy for pain in sickle cell disease: a phase-2 randomized, placebo-controlled trial
10.1002/ajh.27692 · 2025 · External reference
End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings
10.1182/bloodadvances.2019000883 · 2019 · External reference
Gene therapy for sickle cell disease: recent advances, clinical trials and future directions
10.1016/j.jcyt.2024.11.006 · 2025 · External reference
A new frontier: FDA approvals for gene therapy in sickle cell disease
10.1016/j.ymthe.2024.01.015 · 2024 · External reference
Opioid patient controlled analgesia use during the initial experience with the IMPROVE PCA trial: a phase III analgesic trial for hospitalized sickle cell patients with painful episodes
10.1002/ajh.22176 · 2011 · External reference
IMPROVE trial: a randomized controlled trial of patient-controlled analgesia for sickle cell painful episodes: rationale, design challenges, initial experience, and recommendations for future studies
10.1177/1740774513475850 · 2013 · External reference
Current challenges and new approaches to implementing optimal management of sickle cell disease in sub-Saharan Africa
10.1053/j.seminhematol.2023.08.002 · 2023 · External reference
MAGiC: VOC remains but kids with SCA appear
10.1182/blood-2015-08-662502 · 2015 · External reference
N-of-1 studies in an era of precision medicine
10.1001/jama.2024.14637 · 2024 · External reference
A community-centered approach to sickle cell disease and clinical trial participation: an evaluation of perceptions, facilitators, and barriers
10.1182/bloodadvances.2020003434 · 2021 · External reference
Exploring how children and young people with sickle cell disease and their families want to be involved in research: a qualitative study
10.1111/hex.70242 · 2025 · External reference
Prerequisites to support high-quality clinical trials in children and young people
10.1136/archdischild-2019-318677 · 2021 · External reference
Challenges in clinical trials for children and young people
10.1136/archdischild-2019-318676 · 2021 · External reference
The Consortium on Newborn Screening in Africa for sickle cell disease: study rationale and methodology
10.1182/bloodadvances.2022007698 · 2022 · External reference
and the faculty and speakers at the First Pan African Workshop on Newborn Screening, Rabat, Morocco, June 12–14, 2019. Empowering newborn screening programs in African countries through establishment of an international collaborative effort
10.1007/s12687-020-00463-7 · 2020 · External reference
Grandchildren of GRNDaD: Shifts in disease-modifying therapy at the adolescent transition in sickle cell disease
10.1111/bjh.20263 · 2025 · External reference
The WHO genomics program of work for equitable implementation of human genomics for global health
10.1038/s41591-024-03225-x · 2024 · External reference
