The combination of hydroxyurea (HU) with thalidomide decreased the need for transfusions and improved hemoglobin levels among pediatric patients with beta-thalassemia, according to the results of a single-arm study published in the journal Blood Advances.

“Transfusion-related complications and lack of resources in low-to-middle-income countries have led to a search for novel therapies to reduce the need for blood transfusions in patients with β-thalassemia,” the authors wrote in their report. The aim of this study was to determine if the addition of HU to thalidomide could improve outcomes.

The single-arm study treated 135 pediatric patients with HU for 6 months, then the combination of HU plus thalidomide for an additional 6 months. All patients were receiving treatment with HU but demonstrated partial response or a decrease in response and all patients unable to undergo bone marrow transplant. The primary endpoints were number of transfusions and hemoglobin level during HU monotherapy and combination treatment.

Continue Reading

The median age of the cohort was 6 years and 57% were male. The median age at first transfusion was 9 months and the median age at initiation of HU was 1.2 years.

There were 65.9% of patients who achieved a good response to HU plus thalidomide, which was defined as no longer requiring blood transfusion. There were 11.9% of patients who responded, indicating at least a 50% decrease in transfusion volume, and 22.2% who did not respond.

The overall need for blood transfusions significantly decreased after 3 to 6 months of combination therapy (P <.001), including for patients who were considered nonresponders (P =.002).

The baseline median hemoglobin level was 6.5 g/dL, which increased to 8 and 8.7 g/dL at 3 and 6 months, respectively, of combination treatment. After 6 months of combination therapy, 66.7% of patients had a hemoglobin level over 8 g/dL.

There were 28.1% of patients who developed an adverse event (AE), all of which resolved with supportive care or dose interruption. The most common AEs were neutropenia or leukopenia and jaundice.

The authors concluded that “the combination therapy demonstrated promising results and could be considered for a diverse patient population with β-thalassemia.”


Ansari SH, Ansari I, Wasim M, et al. Evaluation of the combination therapy of hydroxyurea and thalidomide in β-thalassemia. Blood Adv. 2022;6:6162-6168. doi: 10.1182/bloodadvances.2022007031