Safety and the Short-Term Efficacy of Venetoclax Combined with Azacitidine Followed by Cladribine in Children with Refractory/Relapsed Acute Myeloid Leukemia.
10.19746/j.cnki.issn.1009-2137.2023.06.006
- Author:
Wei-Wei DU
1
;
Su-Xiang LIU
1
;
Yi WANG
1
;
Hai-Long HE
1
;
Ai-Lian GUO
1
;
Shao-Yan HU
1
;
Jun LU
2
Author Information
1. Department of Hematology，Children's Hospital of Soochow University, Suzhou 215000, Jiangsu Province, China.
2. Department of Hematology，Children's Hospital of Soochow University, Suzhou 215000, Jiangsu Province, China.E-mail: drlujun_sz@163.com.
- Publication Type:Journal Article
- Keywords:
acute myeloid leukemia;
azacitidine;
cladribine;
pediatric;
refractory/relapsed;
venetoclax
- MeSH:
Child;
Female;
Humans;
Male;
Azacitidine/therapeutic use*;
Cladribine/therapeutic use*;
Retrospective Studies;
Leukemia, Myeloid, Acute/genetics*;
Neutropenia;
Antineoplastic Combined Chemotherapy Protocols/therapeutic use*
- From:
Journal of Experimental Hematology
2023;31(6):1635-1638
- CountryChina
- Language:Chinese
-
Abstract:
OBJECTIVE:To investigate the safety and the short-term efficacy of venetoclax combined with azacitidine followed by cladribine (VAC regimen) in children with refractory/ relapsed acute myeloid leukemia (AML).
METHODS:The clinical data, treatment outcomes, complications, and blood product consumption of 6 children with refractory/relapsed AML treated with VAC regimen in the Children's Hospital of Soochow University from August 2021 to December 2021 were retrospectively analyzed.
RESULTS:Among the 6 children, there were 1 male and 5 females. 5 cases were refractory AML, and 1 case was relapsed AML, which recurred again 16 months after allogeneic hematopoietic stem cell transplantation. 4 children were accompanied by chromosomes or genes that predicted poor prognosis, such as RUNX1, FLT3-ITD, KMT2A exon 2-exon 8 dup, MLL-AF6, 7q-, KMT2A exon 2-exon 10 dup, etc. After received VAC regimen, 4 cases achieved CR+CRi, 1 case achieved PR (only MRD did not relieve, MRD was 0.59%), and 1 case was NR (but the proportion of bone marrow blasts decreased). All 6 patients had grade Ⅳ neutropenia, and 4 patients had grade Ⅳ thrombocytopenia. During the period of neutropenia, none of the 6 children developed symptoms of infection such as fever, cough, and diarrhea. No treatment-related death occurred.
CONCLUSION:Venetoclax combined with azacitidine followed by cladribine provides a new treatment option for patients with relapsed/refractory AML who have poor efficacy in early induction remission theragy, showing good efficacy and safety.
