JMML
Conditions
Brief summary
Juvenile myelomonocytic leukemia (JMML) has a poor prognosis in general, with hematopoietic stem cell transplant (HSCT) remaining the standard of care for cure. Azacitidine and homoharringtonine, which exhibited a anticancer mechanism and has been widely used in patients with relapse/refractory acute myeloid leukemia (AML). However, the efficacy of these agents has not been elucidated in children with JMML. The primary objective is to assess the treatment effect on response rate in this subject population.
Interventions
DRUGAzacitidine and homoharringtonine
Concommitant use of azacitidine and homoharringtonine
Sponsors
Qilu Hospital of Shandong University
Taian City Central Hospital
The Affiliated Hospital of Qingdao University
The Second Hospital of Hebei Medical University
Children's Hospital of Hebei Province
Air Force Military Medical University, China
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Months to 18 Years
Healthy volunteers
No
Inclusion criteria
* Newly diagnosed JMML, with PB and BM confirming diagnosis prior to informed consent signature, with one of the following: 1. somatic mutation in PTPN11 2. somatic mutation in KRAS 3. somatic mutation in NRAS and HbF % \> 5x normal value for age 4. clinical diagnosis of neurofibromatosis Type 1.
Exclusion criteria
* 1\. Any condition that would prevent the subject from participating in the study. 2\. Any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if participated in the study. 3\. Any condition that confounds the ability to interpret data from the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Juvenile Myelomonocytic Leukemia (JMML) response rate at end of 4 Months | Up to 4 Months | Defined as proportion of patients with sustained clinical complete remission \[cCR\] or clinical partial remission \[cPR\] according to the International JMML response criteria in Niemeyer 2014 at 3 months (28 days cycles). Response must be sustained for at least 4 weeks either in the 4-week period preceding or succeeding 3 months (ie, sustained over the period minimum 2 months to end of 3 months, or end of 3 months to end of 4 months). |
Countries
China
Contacts
Primary ContactWen-Yu Yang, M.D.
Outcome results
None listed