Hydroxyurea monotherapy does not yield robust hemoglobin increases in patients with SCD, highlighting a need for combination therapy to effectively treat chronic anemia.
Hydroxyurea monotherapy is insufficient to treat chronic anemia in sickle cell disease (SCD); rather, a combination therapy approach is likely to yield more clinical benefits, according to a study published in Blood RCI
Chronic anemia is a characteristic feature of SCD, but therapies that deal with this specific issue are lacking. Packed red blood cell (RBC) transfusions are often repeatedly prescribed, but their chronic use can lead to secondary problems, such as iron overload.
Researchers presented the findings of a study that investigated how the use of hydroxyurea affected anemia parameters among patients with SCD. This retrospective, single-center study included 82 patients with SCD who were treated under the UPMC Adult Sickle Cell Program. The investigators collected medical records of patients who were started on hydroxyurea between January 2007 and May 2023. Patients who received transfusions within 28 days before hydroxyurea initiation and the 3-month follow-up period were excluded from this study.
The mean age of hydroxyurea initiation was 28 years and 44% were male. All participants (100%) identified as Black or African American. The most common genotype was HbSS (51%), followed by HbSC (33%)
Compared with baseline, hydroxyurea initiation was associated with a 0.4 g/dL increase in hemoglobin at 1 month and a 0.3 g/dL increase at 3 months; this increase was deemed “modest.” A hemoglobin increase by 1 g/dL or more was seen in 26.3% of participants at 1 month and 21.6% of patients at 3 months; this was not deemed “robust.” Similar hemoglobin responses at both time intervals suggested that a steady-state hemoglobin response was achieved during this study’s timeframe.
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This study underlines the lack of existing drug therapies for the treatment of chronic anemia in SCD and argues for the use of combination therapy with [hydroxyurea] to improve anemia-related outcomes in SCD.
The researchers next identified other factors associated with hemoglobin increase. They found that a number of factors were associated with a more favorable increase in hemoglobin levels following hydroxyurea initiation; these included lower baseline hemoglobin, male sex, younger age, and less severe genotype.
“This study underlines the lack of existing drug therapies for the treatment of chronic anemia in SCD and argues for the use of combination therapy with [hydroxyurea] to improve anemia-related outcomes in SCD,” the study authors concluded in their report.
Disclosure: Some study authors may have declared affiliations with biotech, pharmaceutical, or device companies. Please see the original reference for a full list of authors’ disclosures
Saber M, Tukakira JK, Kiriza NK, et al. Hydroxyurea monotherapy is insufficient to treat chronic anemia in sickle cell disease: a retrospective study. Blood RCI.Published online March 20, 2026. doi:10.1016/j.brci.2026.100048
