Indication for HSCT in genetic subgroups of JMML
| . | PTPN11 . | K-RAS . | N-RAS . | NF1 . | CBL . |
|---|---|---|---|---|---|
| Germline mutations (± LOH) | Noonan syndrome | Noonan syndrome | Noonan syndrome | Neurofibromatosis type 1 | CBL syndrome “watch and wait” |
| “watch and wait” (mild chemotherapy) | “watch and wait” (mild chemotherapy) | “watch and wait” (mild chemotherapy) | HSCT | HSCT if disease progression occurs | |
| Somatic mutations | HSCT | HSCT | HSCT for most | — | — |
| . | PTPN11 . | K-RAS . | N-RAS . | NF1 . | CBL . |
|---|---|---|---|---|---|
| Germline mutations (± LOH) | Noonan syndrome | Noonan syndrome | Noonan syndrome | Neurofibromatosis type 1 | CBL syndrome “watch and wait” |
| “watch and wait” (mild chemotherapy) | “watch and wait” (mild chemotherapy) | “watch and wait” (mild chemotherapy) | HSCT | HSCT if disease progression occurs | |
| Somatic mutations | HSCT | HSCT | HSCT for most | — | — |
LOH, loss of heterozygosity; HSCT, hematopoietic stem cell transplantation.