Table 2.
PDX mouse models of MDS.
| Name | Dysplastic lineages | Cytopenias* | Ring sideroblasts as % of marrow erythroid elements | BM and PB blasts | Cytogenetics by conventional karyotype analysis | Engraftment Model |
|---|---|---|---|---|---|---|
| MDS with single lineage dysplasia (MDS-SLD) | 1 | 1 or 2 | <15% or <5% if SF3B1 mutant | BM <5%, PB <1%, no Auer rods | Any, unless fulfills all criteria for MDS with isolated del(5q) | MISTRG (76); NSG/NSG-S (77); NSG (78); NOG (79) |
| MDS with multilineage dysplasia (MDS-MLD) | 2 or 3 | 1-3 | <15% or <5% if SF3B1 mutant | BM <5%, PB <1%, no Auer rods | Any, unless fulfills all criteria for MDS with isolated del(5q) | MISTRG (76); NSG/NSG-S (77, 80); NSG (78) |
| MDS with ring sideroblasts (MDS-RS) | ||||||
| MDS-RS with single lineage dysplasia (MDS-RS-SLD) | 1 | 1 or 2 | ≥15% or ≥5% if SF3B1 mutant | BM <5%, PB <1%, no Auer rods | Any, unless fulfills all criteria for MDS with isolated del(5q) | MISTRG (76); NSG/NSG-S (77, 80); NSG (78, 81) |
| MDS-RS with multilineage dysplasia (MDS-RS-MLD) | 2 or 3 | 1-3 | ≥15% or ≥5% if SF3B1 mutant | BM <5%, PB <1%, no Auer rods | Any, unless fulfills all criteria for MDS with isolated del(5q) | MISTRG (76); NSG/NSG-S (77, 80) |
| MDS with isolated del(5q) | 1-3 | 1-2 | None or any | BM <5%, PB <1%, no Auer rods | del(5q) alone or with 1 additional abnormality except −7 or del(7q) | MISTRG (76); NSG/NSG-S (77, 80) |
| MDS with excess blasts (MDS-EB) | ||||||
| MDS-EB-1 | 0-3 | 1-3 | None or any | BM 5%-9% or PB 2%-4%, no Auer rods | Any | MISTRG (76); NSG/NSG-S (77, 80); NSG (78, 81); NOG (79) |
| MDS-EB-2 | 0-3 | 1-3 | None or any | BM 10%-19% or PB 5%-19% or Auer rods | Any | MISTRG (76); NSG/NSG-S (77); NSG (78); NOG (79) |
| MDS, unclassifiable (MDS-U) | NSG (78); NSG/NSG-S (80) | |||||
| with 1% blood blasts | 1-3 | 1-3 | None or any | BM <5%, PB = 1% on 2 separate occasions; no Auer rods | Any | |
| with single lineage dysplasia and pancytopenia | 1 | 3 | None or any | BM <5%, PB <1%, no Auer rods | Any | |
| based on defining cytogenetic abnormality | 0 | 1-3 | <15% | BM <5%, PB <1%, no Auer rods | MDS-defining abnormality | |
| Refractory cytopenia of childhood | 1-3 | 1-3 | None | BM <5%, PB <2% | Any | |
*Cytopenias defined as: hemoglobin, <10 g/dL; platelet count, <100 × 109/L; and absolute neutrophil count, <1.8 × 109/L. Rarely, MDS may present with mild anemia or thrombocytopenia above these levels. PB monocytes must be <1 × 109/L.
Song et al. performed targeted exome sequencing to validate engraftment of clonal MDS.
Pang et al. and Muguruma et al. performed FISH for monosomy 7 to validate engraftment of clonal MDS.
Muguruma et al. and Meydouf et al. used autologous or allogenetic MSC to enhance MDS engraftment.