Bone marrow failure disease
Conditions
Interventions
None listed
Sponsors
National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
Eligibility
Sex/Gender
All
Age
No minimum to 18 Years
Inclusion criteria
Inclusion criteria: 1. Age at diagnosis < 18 years; 2. From 2011.1.1, diagnosed in the sub-centers and meet the following disease diagnostic criteria: (1) IBMFs sub-cohort: Diagnosed and highly suspected inherited bone marrow failure disease. Including Fanconi anemia, Diamond-Blackfan anemia, Dyskeratosis Congenita, Shwachman-Diamond syndrome, congenital neutropenia, bone marrow failurerelated congenital thrombocytopenia, and Unclassifiable Inherited bone marrow failure syndrome; (2) MDS and MDS-related disease sub-cohort: highly suspected MDS, MDS, and AML-MRC, including primary MDS and secondary MDS which progressed from IBMFs; (3) Sever aplastic anemia sub-cohort: the patients diagnosed with SAA; (4) Transplantation-treated bone marrow failure sub-cohort: the patients who were diagnosed with bone marrow failure disease and underwent transplantation; Patients with difficulty in diagnosis are regularly discussed by experts from the scientific committee to determine whether they can be included in the group.
Exclusion criteria
Exclusion criteria: 1. Bone marrow failure (pancytopenia)secondary to non-hematopoietic disease(patients with hepatitis associated aplastic anemia can be included).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Remission rate;Overall survival;Blood cell count; | — |
Countries
China
Contacts
Public ContactZhu Xiaofan
National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
Outcome results
None listed