Clinical features recorded by medical record review
| Data type . | Specific variables recorded . |
|---|---|
| Genetic/cytogenetic abnormalities∗ | ASXL1, TET2, RUNX1, SETBP1, DNMT3A, U2AF1, EZH2, TP53, BCOR, BCORL1, EZH2, CUX1, del(13q), −Y, +8, del 7/7q, complex karyotype, germ line testing for inherited causes of AA (see the supplemental Methods for full list) |
| Blood counts† | Hgb, Plt, ALC, ANC, IPF, MCV, ARC, RC |
| PNH clone | PNH flow cytometry date, PNH clone distribution (RBC type 2, type 3, total), monocyte, granulocyte, initial PNH clone, secondary PNH clone |
| Demographic information | Age at definitive AA diagnosis, age at sMN diagnosis, sex |
| Follow-up information | Date of last appointment, date of death, date of initial diagnosis, survival time after diagnosis, survival time after sMN |
| Clonal evolution event information | Date of sMN diagnosis, sMN type (AML, MDS), time elapsed between AA diagnosis and sMN evolution |
| Treatment information | Treatment types (antithymocyte globulin, cyclosporine, eltrombopag, HSCT), treatment start date(s), response (NR/PR/CR§), time elapsed between diagnosis and treatment type, time elapsed between treatment and sMN evolution |
| Miscellaneous | AST, ALT, ferritin, bone marrow cellularity (%), aplastic anemia disease severity‡ |
| Data type . | Specific variables recorded . |
|---|---|
| Genetic/cytogenetic abnormalities∗ | ASXL1, TET2, RUNX1, SETBP1, DNMT3A, U2AF1, EZH2, TP53, BCOR, BCORL1, EZH2, CUX1, del(13q), −Y, +8, del 7/7q, complex karyotype, germ line testing for inherited causes of AA (see the supplemental Methods for full list) |
| Blood counts† | Hgb, Plt, ALC, ANC, IPF, MCV, ARC, RC |
| PNH clone | PNH flow cytometry date, PNH clone distribution (RBC type 2, type 3, total), monocyte, granulocyte, initial PNH clone, secondary PNH clone |
| Demographic information | Age at definitive AA diagnosis, age at sMN diagnosis, sex |
| Follow-up information | Date of last appointment, date of death, date of initial diagnosis, survival time after diagnosis, survival time after sMN |
| Clonal evolution event information | Date of sMN diagnosis, sMN type (AML, MDS), time elapsed between AA diagnosis and sMN evolution |
| Treatment information | Treatment types (antithymocyte globulin, cyclosporine, eltrombopag, HSCT), treatment start date(s), response (NR/PR/CR§), time elapsed between diagnosis and treatment type, time elapsed between treatment and sMN evolution |
| Miscellaneous | AST, ALT, ferritin, bone marrow cellularity (%), aplastic anemia disease severity‡ |
ALC, absolute leukocyte count; ALT, alanine aminotransferase ANC, absolute neutrophil count; ARC, absolute reticulocyte count; AST, aspartate aminotransferase; CR, complete response; Hgb, hemoglobin; IPF, immature platelet fraction; MCV, mean corpuscular volume; NR, no response; Plt, platelet count; PR, partial response; RC%, reticulocyte count percentage.
Data obtained from the initial diagnostic workup and at sMN diagnosis. Recorded as VAF (%) of mutation or as presence/absence of mutation.
Data obtained from the initial diagnostic workup and at the 6-month post-IST assessment.
As defined by the Camitta criteria19.
As defined by the National Institutes of Health criteria20,21.