Table 2Summary of Evidence About the Toxicity of Hydroxyurea in Sickle Cell Disease*

OutcomesEvidence GradeBasis for Grade
Key Question 3—Children
Leukemia (MDS/AML/Cytogenetic abnormalities)InsufficientCERHR report
Developmental toxicities (in utero)Evidence of harm in animalsCERHR report
Leg ulcersInsufficientCERHR report
Growth delaysEvidence of no growth delayCERHR report
Developmental toxicities in next generationInsufficientCERHR report
Key Question 3—Adults
Leukemia (MDS/AML/Cytogenetic abnormalitiesLowIndirect evidence and inconsistent results
Leg ulcersHigh (absence of effect)One good RCT, plus consistent observational studies
Skin neoplasmsInsufficientNo studies in sickle cell; high-grade evidence in other populations
Secondary malignanciesInsufficientNo studies in sickle cell; low-grade evidence in other populations
Adverse pregnancy outcomesInsufficientCEHER report
Spermatogenesis defectsLowCase reports with evidence of causality

Evidence grades as on Table 1;

MDS = myelodysplastic syndromes; AML = acute myelogenous leukemia; CEHER = Center for the Evaluation of Risks to Human Reproduction.

From: Executive Summary

Cover of Hydroxyurea for the Treatment of Sickle Cell Disease
Hydroxyurea for the Treatment of Sickle Cell Disease.
Evidence Reports/Technology Assessments, No. 165.
Segal JB, Strouse JJ, Beach MC, et al.

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