This research is being done to study the efficacy and safety of unrelated umbilical cord blood stem cell microtransplantation combined with azacitidine(AZA) based treatment for advanced myelodysplastic syndromes(MDS), Chronic myelomonocytic leukemia-2(CMML-2) and secondary acute myeloid leukemia(sAML). The study protocol involved unrelated umbilical cord blood stem cell combined with azacitidine based treatment, which including azacitidine alone and azacitidine plus a targeted agent or chemotherapy agent.
MDS patients diagnosed due to bone marrow morphology and immunophenotyping (met the World Health Organization(WHO) 2016 diagnosis criterion) and met revised the International Prognostic Scoring System(IPSS) prognostic risk categories intermediate(>3-≤4.5), high(>4.5-≤6), and very high risk(>6), or CMML-2(PB 5-19% or bone marrow 10-19% blast equivalent, and/or when any Auer rods are present) patients diagnosed due to bone marrow morphology and immunophenotyping (met the WHO 2016 diagnosis criterion) with white blood cell count(WBC)<13x109/L, or AML patients diagnosed due to bone marrow morphology and immunophenotyping (met the WHO 2016 diagnosis criterion) with history of myeloid neoplasms ;
Ineligible for allogeneic hematopoietic stem cell transplantation (HSCT) based on local medical standards and clinical decision guidelines in the study;
Indications for azacytidine (AZA) treatment were determined according to local medical standards and diagnostic decision guidelines in the study;
Morphology and immunophenotyping excluded BCR/ABL positive chronic myelogenous leukemia and other myeloid neoplasms including essential thrombocythemia, polycythemia vera and primary myelofibrosis;
BCR/ABL positive chronic myelogenous leukemia, primary myelofibrosis, polycythemia vera and essential thrombocythemia;
Low risk myelodysplastic syndromes according to IPSS or revised IPSS prognostic risk categories;
Acute promyelocytic leukemia, myeloid sarcoma, or accelerated or blastic phase of chronic myelogenous leukemia;
Allergic to any of the mentioned agent in the study protocol;
Single group
unrelated umbilical cord blood
DrugA single unit of unrelated umbilical cord blood was reinfused within 24-72 hours after the end of AZA or chemotherapy, and the longest delay was 96 hours after the end of chemotherapy. The umbilical cord blood was matched at 0-3/10 locus. Microtransplantation of umbilical cord blood: single unit of unrelated cord blood (HLA 0-5/10 or 0-3/6 matched, TNC 1-2×107/Kg body weight), AZA or homoharringtonine infusion 24-72 hours after the end of injection.
overall survival rate
It is measured from the date of randomization to the date of death from any cause; patients not known to have died at least follow-up are censored on the date they were last known to be alive.
complete response rate
Complete remission was achieved according to the remission criteria of different diseases Complete remission was achieved according to the remission criteria of different diseases Complete remission was achieved according to the remission criteria of different diseases Complete remission was achieved according to the remission criteria of different diseases complete response rate is achieved according to the remission criteria of different diseases
Hematopoietic function recovery time
The recovery time of hematopoietic function was defined as the time from the decline of three lineages to the stable hematological response of three lineages
The 2-year overall survival rate
The 2-year survival rate was calculated as the number of surviving cases/the total number of cases followed up for 2 years
disease-free survival
The disease-free survival was calculated as the time from randomization to disease recurrence or death due to disease progression
Early mortality
Early mortality was defined as death within the first 3 months after initiation of induction therapy
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