A phase 2 clinical trial of luspatercept in non-transfusion-dependent patients with myelodysplastic syndromes

Luspatercept has shown durable clinical efficacy for the treatment of anemia in transfusion-dependent patients with lower-risk myelodysplastic syndromes (LR-MDS). We report the results of a prespecified primary analysis of a phase 2 trial of luspatercept in non-transfusion-dependent (NTD) Japanese patients with anemia due to LR-MDS. Luspatercept (starting dose 1.0 mg/kg) was administered subcutaneously once every 3 weeks. The primary endpoint was the proportion of patients who achieved hematological improvement-erythroid (HI-E) response (≥ 1.5 g/dL increase in hemoglobin level for 8 weeks) without transfusions within the first 24 weeks of treatment. At the primary analysis data cutoff, 21 patients had been enrolled/treated; 17 and 10 patients had completed 24 and 48 weeks of treatment, respectively. HI-E response occurred within 24 weeks in 10 patients (47.6%; 95% confidence interval, 25.7-70.2; P < 0.0001), which was significantly higher than the predefined threshold (10%). By week 48, HI-E response occurred in 12 patients (57.1%) and 17 patients (81.0%) remained NTD. Luspatercept was well tolerated. Three patients (14.3%) had grade 3-4 treatment-related treatment-emergent adverse events. Luspatercept resulted in statistically and clinically significant improvements in hemoglobin levels, and may help delay the need for transfusions in NTD patients with LR-MDS.

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Publication Details

Journal
International Journal of Hematology
Published
2024-11-21
DOI
https://doi.org/10.1007/s12185-024-03872-3
Citations
2
Primary Topic
Acute Myeloid Leukemia Research
Type
article
Field-Weighted Citation Impact
0.91

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article

A phase 2 clinical trial of luspatercept in non-transfusion-dependent patients with myelodysplastic syndromes

Shiro Kubonishi, Hiroatsu Iida, Atsushi Shinagawa, Yasuyoshi Morita et al.
2 citations
International Journal of Hematology
Acute Myeloid Leukemia Research
0.91
article

A phase 2 clinical trial of luspatercept in non-transfusion-dependent patients with myelodysplastic syndromes

Shiro Kubonishi, Hiroatsu Iida, Atsushi Shinagawa, Yasuyoshi Morita, Yasuhiro Nakashima, Kenshi Suzuki, Shuichi Midorikawa, Kensuke Usuki, Tomoaki Fujisaki, Makoto Suda, Tatsuro Jo, J. Hayakawa, Takahiro Suzuki, Hiromi Iwasaki, Kosei Matsue, Hideyuki Yonaga, Akira Yokota, Mitsufumi Nishio, Hiroshi Kosugi, Yotaro Tamai, Koichi Onodera
article en
2 citations

Abstract

Luspatercept has shown durable clinical efficacy for the treatment of anemia in transfusion-dependent patients with lower-risk myelodysplastic syndromes (LR-MDS). We report the results of a prespecified primary analysis of a phase 2 trial of luspatercept in non-transfusion-dependent (NTD) Japanese patients with anemia due to LR-MDS. Luspatercept (starting dose 1.0 mg/kg) was administered subcutaneously once every 3 weeks. The primary endpoint was the proportion of patients who achieved hematological improvement-erythroid (HI-E) response (≥ 1.5 g/dL increase in hemoglobin level for 8 weeks) without transfusions within the first 24 weeks of treatment. At the primary analysis data cutoff, 21 patients had been enrolled/treated; 17 and 10 patients had completed 24 and 48 weeks of treatment, respectively. HI-E response occurred within 24 weeks in 10 patients (47.6%; 95% confidence interval, 25.7-70.2; P < 0.0001), which was significantly higher than the predefined threshold (10%). By week 48, HI-E response occurred in 12 patients (57.1%) and 17 patients (81.0%) remained NTD. Luspatercept was well tolerated. Three patients (14.3%) had grade 3-4 treatment-related treatment-emergent adverse events. Luspatercept resulted in statistically and clinically significant improvements in hemoglobin levels, and may help delay the need for transfusions in NTD patients with LR-MDS.

International Journal of HematologyVol. 121(1)
Japanese Red Cross Society, Japan (JP), NTT Medical Center (JP), National Kyushu Medical Center (JP), Tohoku University Hospital (JP), Kindai University Hospital (JP), Japanese Red Cross Medical Center (JP), Kitasato University Hospital (JP), Kameda Medical Center (JP), Matsuyama Red Cross Hospital (JP), Ogaki Municipal Hospital (JP), Himeji Red Cross Hospital (JP), Chiba Aoba Municipal Hospital (JP), National Hospital Organization (JP), Bristol-Myers Squibb (Japan) (JP), Hitachi General Hospital (JP), Shonan Kamakura General Hospital (JP), Nagoya Medical Center (JP), Osaka Metropolitan University (JP)
Bristol-Myers Squibb
Good health and well-being
Openalex Percentile: Top 22%
Acute Myeloid Leukemia Research
0.91
DOIOpen Access PDFOpenAlex
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