Scholarlink

Background <p>Acute myeloid leukemia (AML) with persistent measurable residual disease (MRD) and relapsed/refractory myelodysplastic syndromes (MDS) are low-blast myeloid diseases for which there are few effective therapeutic options. CD123 represents an attractive target in these diseases. Vibecotamab is a bispecific antibody that binds to CD123 on malignant blasts and to CD3 on T-cells, to recognize and eliminate CD123-positive malignant cells.</p> Methods <p>This single-center phase II study evaluated the efficacy of vibecotamab in patients AML with detectable MRD (AML-MRD cohort) or with MDS or chronic myelomonocytic leukemia (CMML) after hypomethylating agent failure (MDS/CMML cohort). In cycle 1, patients received vibecotamab IV on day 1 (0.43&#xa0;µg/kg), day 3 (0.75&#xa0;µg/kg), day 5 (1.1&#xa0;µg/kg), and days 8, 15 and 22 (1.7&#xa0;µg/kg). In subsequent cycles, patients received vibecotamab IV on days 1, 8, 15, and 22 (1.7&#xa0;µg/kg). The primary outcomes were MRD negativity rate (AML-MRD cohort) and overall response (MDS/CMML cohort).</p> Results <p>Between May 2022 and April 2025, 48 patients were enrolled (21 AML-MRD cohort, 27 MDS/CMML cohort). The median ages of the AML-MRD and the MDS/CMML cohorts were 70 and 76 years, respectively. In the AML-MRD cohort, the median MRD level by flow cytometry was 0.64% (range, 0.1–3.9%), and in the MDS/CMML, the median bone marrow blast percentage was 7% (range, 3–19%). The AML-MRD clearance rate was 19% (4/21; 95% CI 5–42%), and in the MDS/CMML cohort, the overall response rate was 67% (18/27; 95% CI 46–83%). The median overall survival was 13.1 months (95% CI 8.9-NR) for the AML-MRD cohort and 6.5 months (95% CI 4.2–10.3) for the MDS/CMML cohort. The most frequent adverse event was infusion reaction or cytokine relapse syndrome, which occurred in 29 patients (60%) overall, most of which were grade 1–2.</p> Conclusions <p>Vibecotamab was active in low-blast myeloid diseases, although the durability of responses was modest. Additional studies of CD123-targeting bispecific antibodies, alone or in combination, are warranted for these diseases.</p> Trial registration <p>Clinicaltrials.gov (NCT05285813).</p>

错误：搜索内容不能为空，请输入英文关键词
错误：关键词超出字数限制，请精简
高级检索

Vibecotamab for measurable residual disease in acute myeloid leukemia and for myelodysplastic syndromes and chronic myelomonocytic leukemia after hypomethylating agent failure: a phase II study

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2026年6月27日

查看全文
  • Nicholas J. Short,
  • Alex Bataller,
  • Courtney D. DiNardo,
  • Guillermo Montalban-Bravo,
  • Sa A. Wang,
  • Wei Wang,
  • Abhishek Maiti,
  • Daniel Nguyen,
  • Maria C. Hachem,
  • Timothy M. Bi,
  • Ghayas C. Issa,
  • Kelly S. Chien,
  • Maro Ohanian,
  • Tapan M. Kadia,
  • Sanam Loghavi,
  • Omer Karrar,
  • Pavan Bachireddy,
  • Hyunsoo Hwang,
  • Xuelin Huang,
  • Guillermo Garcia-Manero,
  • Farhad Ravandi

摘要

Background

Acute myeloid leukemia (AML) with persistent measurable residual disease (MRD) and relapsed/refractory myelodysplastic syndromes (MDS) are low-blast myeloid diseases for which there are few effective therapeutic options. CD123 represents an attractive target in these diseases. Vibecotamab is a bispecific antibody that binds to CD123 on malignant blasts and to CD3 on T-cells, to recognize and eliminate CD123-positive malignant cells.

Methods

This single-center phase II study evaluated the efficacy of vibecotamab in patients AML with detectable MRD (AML-MRD cohort) or with MDS or chronic myelomonocytic leukemia (CMML) after hypomethylating agent failure (MDS/CMML cohort). In cycle 1, patients received vibecotamab IV on day 1 (0.43 µg/kg), day 3 (0.75 µg/kg), day 5 (1.1 µg/kg), and days 8, 15 and 22 (1.7 µg/kg). In subsequent cycles, patients received vibecotamab IV on days 1, 8, 15, and 22 (1.7 µg/kg). The primary outcomes were MRD negativity rate (AML-MRD cohort) and overall response (MDS/CMML cohort).

Results

Between May 2022 and April 2025, 48 patients were enrolled (21 AML-MRD cohort, 27 MDS/CMML cohort). The median ages of the AML-MRD and the MDS/CMML cohorts were 70 and 76 years, respectively. In the AML-MRD cohort, the median MRD level by flow cytometry was 0.64% (range, 0.1–3.9%), and in the MDS/CMML, the median bone marrow blast percentage was 7% (range, 3–19%). The AML-MRD clearance rate was 19% (4/21; 95% CI 5–42%), and in the MDS/CMML cohort, the overall response rate was 67% (18/27; 95% CI 46–83%). The median overall survival was 13.1 months (95% CI 8.9-NR) for the AML-MRD cohort and 6.5 months (95% CI 4.2–10.3) for the MDS/CMML cohort. The most frequent adverse event was infusion reaction or cytokine relapse syndrome, which occurred in 29 patients (60%) overall, most of which were grade 1–2.

Conclusions

Vibecotamab was active in low-blast myeloid diseases, although the durability of responses was modest. Additional studies of CD123-targeting bispecific antibodies, alone or in combination, are warranted for these diseases.

Trial registration

Clinicaltrials.gov (NCT05285813).