Trial reportAmerican journal of hematology2010
The risks and benefits of long-term use of hydroxyurea in sickle cell anemia: A 17.5 year follow-up.
Trial report in American journal of hematology, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06199557 (A Phase 1/2 Multicenter Open-label Study to Investigate Treatment of Hydroxyurea in Combination With Valproic Acid), which is not on this map. Cited by 232 papers, 15 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase 1/2 Multicenter Open-label Study to Investigate Treatment of Hydroxyurea in Combination With Valproic Acid (VPA), or 6- Mercaptopurine in Combination With VPA in Patients With AML or HR-MDS Unfit for Standard Therapy
Who cites it
232 citing papers in PubMed, 15 syntheses or guidelines pooled it, 477 citations in OpenAlex.
- Risk of cancer in patients with thalassemia and sickle cell disease: a systematic review.Annals of medicine · 2025Pooled it
- Foetal haemoglobin inducers for reducing blood transfusion in non-transfusion-dependent beta-thalassaemias.The Cochrane database of systematic reviews · 2023Pooled it
- Hydroxyurea (hydroxycarbamide) for sickle cell disease.The Cochrane database of systematic reviews · 2022Pooled it
- Pooled it
- Phytomedicines (medicines derived from plants) for sickle cell disease.The Cochrane database of systematic reviews · 2020Pooled it
- Hematopoietic stem cell transplantation for people with sickle cell disease.The Cochrane database of systematic reviews · 2020Pooled it
- Interventions for preventing silent cerebral infarcts in people with sickle cell disease.The Cochrane database of systematic reviews · 2020Pooled it
- Phytomedicines (medicines derived from plants) for sickle cell disease.The Cochrane database of systematic reviews · 2018Pooled it
- Interventions for preventing silent cerebral infarcts in people with sickle cell disease.The Cochrane database of systematic reviews · 2017Pooled it
- Hydroxyurea (hydroxycarbamide) for sickle cell disease.The Cochrane database of systematic reviews · 2017Pooled it
- Risk factors for mortality in adult patients with sickle cell disease: a meta-analysis of studies in North America and Europe.Haematologica · 2017Pooled it
- Hydroxyurea for reducing blood transfusion in non-transfusion dependent beta thalassaemias.The Cochrane database of systematic reviews · 2016Pooled it
- Systematic review of interventional sickle cell trials registered in ClinicalTrials.gov.Clinical trials (London, England) · 2015Pooled it
- A systematic review of known mechanisms of hydroxyurea-induced fetal hemoglobin for treatment of sickle cell disease.Expert review of hematology · 2015Pooled it
- An official American Thoracic Society clinical practice guideline: diagnosis, risk stratification, and management of pulmonary hypertension of sickle cell disease.American journal of respiratory and critical care medicine · 2014Guideline
- Hematopoietic cell transplant compared with standard care in adolescents and young adults with sickle cell disease.Blood advances · 2025Trial
- Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease.Scientific reports · 2024Trial
- Early initiation of hydroxyurea (hydroxycarbamide) using individualised, pharmacokinetics-guided dosing can produce sustained and nearly pancellular expression of fetal haemoglobin in children with sickle cell anaemia.British journal of haematology · 2021Trial
- Non-myeloablative human leukocyte antigen-matched related donor transplantation in sickle cell disease: outcomes from three independent centres.British journal of haematology · 2021Trial
- Hydroxyurea for Children with Sickle Cell Anemia in Sub-Saharan Africa.The New England journal of medicine · 2019Trial
172 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
12 authors at 11 institutions in 1 country.
Funding
Abstract
A randomized, controlled clinical trial established the efficacy and safety of short-term use of hydroxyurea in adult sickle cell anemia. To examine the risks and benefits of long-term hydroxyurea usage, patients in this trial were followed for 17.5 years during which they could start or stop hydroxyurea. The purpose of this follow-up was to search for adverse outcomes and estimate mortality. For each outcome and for mortality, exact 95% confidence intervals were calculated, or tests were conducted at alpha = 0.05 level (P-value <0.05 for statistical significance). Although the death rate in the overall study cohort was high (43.1%; 4.4 per 100 person-years), mortality was reduced in individuals with long-term exposure to hydroxyurea. Survival curves demonstrated a significant reduction in deaths with long-term exposure. Twenty-four percent of deaths were due to pulmonary complications; 87.1% occurred in patients who never took hydroxyurea or took it for <5 years. Stroke, organ dysfunction, infection, and malignancy were similar in all groups. Our results, while no longer the product of a randomized study because of the ethical concerns of withholding an efficacious treatment, suggest that long-term use of hydroxyurea is safe and might decrease mortality.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.