Myelodysplastic syndrome
Conditions
Interventions
RCC Group:none
RCC-T Group:Hematopoietic stem cell transplantation(HSCT)
RAEB-HAMs-HSCT Group :Transplant after chemotherapy with demethylating drugs(HAMs+HSCT)
RAEB-HSCT Group:Hematopoietic stem cell transplantation(HSCT)
RAEBT-HAMs-HSCT Group:Transplant after chemotherapy with demethylating drugs(HAMs+HSCT)
RAEBT-HAMs-LDC-HSCT Group:Transplantation after demethylation drugs combined with low-dose chemotherapy(HAMs+LDC+HSCT)
Sponsors
Children's Hospital of Soochow University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Children, younger than 18 years old; 2. MDS is accompanied by a specific karyotype, such as -7/7q-, +8, +20 for more than 3 months; 3. Peripheral blood and bone marrow immature cells conform to MDS-RAEB and MDS-RAEBt; 4. MDS-specific gene mutations, such as GATA2, RUNX1, RNKRD26, ETV6, SAMD9/SAMDL mutations that occur in germline; 5. Familial MDS; 6. FA (Fanconi anemia), DBA (congenital pure red aplastic anemia), SCN (congenital agranulocytosis), SDS (Shu-Dai syndrome), DC (congenital dyskeratosis), AT (congenital dyskeratosis) Thrombocytopenia without megakaryocyte production) secondary MDS; 7. MDS secondary to Down syndrome; 8. MDS secondary to any other congenital diseases; 9. MDS secondary to severe aplastic anemia; 10. Treatment-related MDS; 11. Obtain the consent of the child or/and guardian and sign the informed consent form; 12. The estimated survival time is greater than 3 months.
Exclusion criteria
Exclusion criteria: 1. More than one line of blood cells decreased, with AML characteristic genetic changes; 2. Acquired aplastic anemia; 3. Acquired immune dysfunction diseases; 4. Juvenile myelomonocytic leukemia (JMML); 5. Only do temporary chemotherapy, radiotherapy or immunotherapy, and do not accept systemic treatment in accordance with the treatment plan; 6. Have any significant abnormal coexisting diseases or mental illnesses that affect the patient's life safety and compliance, affect informed consent, research participation, follow-up or result interpretation; 7. Those who have very poor nutritional status, severe infection, cardiac insufficiency, and cannot tolerate treatment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| remission state;Neutrophil implantation time;Platelet implantation time;Percentage of immature cells;Fit degree;Acute / chronic graft versus host disease; | — |
Secondary
| Measure | Time frame |
|---|---|
| Bronchitis obliterans; | — |
Countries
China
Contacts
Public ContactHu Shaoyan
Children's Hospital of Soochow University
Outcome results
None listed