TABLE 2

Toxicities of Hydroxyurea Reported in Pediatric Studies

Authorn (Patient-Years)NeutropeniaPlatelets (<80 000/μL)AnemiaRash/Nail ChangesOther
Ferster et al2025020No clinically significant toxicity; no thrombocytopenia in placebo arm
(12.5)
Kinney et al288456, ANC < 2000/μL7275No creatinine elevation; 12 headache; 11 ALT elevation
Zimmerman et al37122HbSC 3/7∼10%No increase in VDJ rearrangements; 2 deaths (pneumococcal sepsis, acute hemolytic transfusion reaction)
(455)HbSBthal 1/7
Wang et al332817, ANC < 1500/μL 6, ANC < 500/μL172 accidental overdoses; 1 ALT elevation; 1 death (splenic sequestration)
Hankins et al272110 in year 32 in year 53 in year 31 splenic sequestration; 1 death (pneumococcal sepsis)
9 in year 41 in year 61 in year 4
de Montalembert et al24351151 renal failure (from systemic lupus erythematosus); 1 secondary amenorrhea
< 100 000/μL
de Montalembert et al381015, ANC < 1500/μL; 2, ANC < 1000/μL483 headache; 1 leukemia after 1.5 mo; 1 leg ulcer
< 100 000/μL
de Montalembert et al232259916 hypersplenism; 1 azoospermia; 1 leg ulcer (reported earlier)38; 1 leukemia (reported earlier)38; 1 death (asystolic cardiac arrest)
Gulbis et al2610925 < 60 g/L1 leukemia after 6 y; 1 death (splenic sequestration)
(426)< 100 000/μL
Scott et al2115003/1311 death (intracranial hemorrhage); 1 headache
Olivieri and Vichinsky3017930111 creatinine increase; 1 dyspepsia; 1 conjunctivitis
Hanft et al3938HPRT cloning efficiency and VDJ recombination events (see text)
  • ALT indicates alanine aminotransferase; HbSC, sickle-hemoglobin C disease; HbSBthal, sickle β -thalassemia; —, data were not reported.