Juvenile myelomonocytic leukemia
Conditions
Interventions
After diagnosis of JMML and enrollment in the JMML-20 study, azacitidine therapy is started.
Azacitidine is administered as a 10-minute IV infusion of 75 mg/m2 once a day for 7 days (days 1-7), follow
Sponsors
Koh Katsuyoshi
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: (1) Cases with a clinical diagnosis of JMML based on the JMML diagnostic criteria. (2) Have submitted or are able to submit samples for central testing. (3) Consent for CHM-14 was obtained prior to obtaining consent to participate in this study. (4) Age at diagnosis is less than 10 years old. (5) The patient has sufficient organ function to meet the following three criteria at the same time. Laboratory values within 7 days before the date of enrollment. a) D-Bil level: less than 1.5 mg/dL b) Creatinine: less than 3 times the upper limit of the age-specific test reference value c) Left ventricular ejection fraction (EF) >=50% by echocardiography (6) Adequate explanations and written consent have been obtained from the proxy.
Exclusion criteria
Exclusion criteria: (1) Noonan syndrome (a case of PTPN11 germ cell mutation) (2) Associated with difficult-to-control infections (including active tuberculosis and HIV antibody-positive cases). (3) Relapse cases with a history of hematopoietic cell transplantation. (4) Any other case deemed inappropriate by the doctor in charge.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Clinical response rate (cCR + cPR) after 3 cycles of azacitidine therapy | — |
Secondary
| Measure | Time frame |
|---|---|
| Adverse Events (AEs) 1-year, 3-year and 5-year overall survival rates Causes of death 1-year, 3-year and 5-year transplant-free survival rates 1-year, 3-year and 5-year relapse-free survival rates Clinical response at each cycle Genetic response for each cycle Assessment of quality of life of the affected child obtained from a questionnaire by the individual and family (proxy assessment) 5 years after enrollment. Quality of life assessment of the affected child obtained from the physician. Percentage of hematopoietic cell transplants performed. Conditioning regimen for hematopoietic cell transplantation Time to first hematopoietic cell transplantation Engraftment rate after initial hematopoietic cell transplantation Leukemia-free survival after hematopoietic cell transplantation (time from initial hematopoietic cell transplantation to progression of primary disease or death) Overall survival after hematopoietic cell transplantation (time from initial hematopoietic cell transplantation to death) Time to recurrence after hematopoietic cell transplantation (time from initial hematopoietic cell transplantation to recurrence) Time to transplantation-related death after hematopoietic cell transplantation (time from initial hematopoietic cell transplantation to transplantation-related death) Cumulative incidence of post-transplant acute GVHD (grades II - IV) Cumulative incidence of post-transplant acute GVHD (grades III-IV) Cumulative Incidence of chronic GVHD after transplantation Cumulative recurrence rate at 3 years after transplantation Cumulative transplant-related complication mortality at 3 years after transplant 3-year GVHD-free recurrence-free survival after transplantation | — |
Contacts
Public ContactHideki Muramatsu
Nagoya University Hospital
Outcome results
None listed