Real-world data on efficacy and safety of azacitidine therapy in chronic myelomonocytic leukemia in China: results from a multicenter, retrospective study

Abstract

SummaryChronic myelomonocytic leukemia (CMML) is a rare and aggressive myeloid malignancy with overlapped features of myelodysplastic syndromes/myeloproliferative neoplasms. Azacitidine (AZA), a hypomethylating agent, has been approved for the treatment of CMML in China, but real-world data are limited. Medical records of CMML patients who had received subcutaneously injected AZA were reviewed from January 2018 at five participating sites in China. Response was assessed according to the modified International Working Group (IWG 2006) criteria. Between January 2018 and November 2020, a total of 24 patients with CMML were included with a median age of 63 years. Patients received a median of 3 cycles of AZA treatment (range, 1–8). Overall response rate (ORR) was 37.5% (9 of 24); CR rate, PR rate, and mCR/HI rate were 8.3% (n = 2), 8.3% (n = 2), and 20.8% (n = 5), respectively. At a median duration of follow-up of 14.0 months (range 0.0–22.0 months), the median overall survival (OS) was 23.0 months. Univariate analysis revealed that ≥ 3 cycles of treatment was significantly associated with a higher 1-year OS rate compared with < 3 cycles of AZA treatment. Treatment was generally well-tolerated. The most common (> 10%) AEs were thrombocytopenia (n = 7, 29.2%), pneumonitis (n = 4, 16.7%) and fever (n = 3, 12.5%). This study provides valuable real-life data in China on the treatment schedules, efficacy and safety of AZA in the treatment of CMML.

Impact of a novel prognostic model on allogeneic hematopoietic stem cell transplantation outcomes in patients withCMML

“…According to CMML epidemiological statistics, almost 90% of cases occur in patients older than 60 years 4,35,50 . We reviewed the literatures on the age of CMML patients in Western (Germany, 29,30,51 France, 13,19,52 America, 8,11,12,15,17,20,25,26,28,40,53,54 Italy, 39,55 Greece, 14 and Denmark, 24 ) and Eastern countries (Japan 16,22,27 and China, 6,43,49,56–63 ), and the results showed that the median age of CMML patients in Eastern countries was younger (38–70 years vs. 44–73 years), and the median age of CMML patients undergoing allo‐HSCT was similar in Eastern and Western countries (38–63 years vs. 44–64 years). However, the age of CMML patients undergoing allo‐HSCT was lower in both Eastern and Western countries.…”

3055 – Multi-Omics Reveals Azacitidine Partially Restores the Hematopoiesis-Supportive Functions in Mesenchymal Stromal Cells From Patients With Chronic Myelomonocytic Leukemia

“…The two FDA-approved de-methylating compounds for CMML, azacitidine (AZA) and decitabine (DEC) were initially designed to restore the disordered epigenetics in malignant HSPCs, which were thought to be disease-promoting consequences of genetic lesions involving epigenetics (2). However, demonstrated by the limited inhibitory effect on malignant clones in patients, HMAs may not produce e cacies prominently through the direct anti-tumor effect (7)(8)(9)(10). Those post-HMA therapeutic responses (e.g., hematological improvement) (11) are more likely attributed to the selective promotion of residual healthy hematopoiesis in the bone marrow.…”