Management of cytopenia, iron overload, circulating blasts, and splenomegaly
| Condition . | Association with outcome . | Management . |
|---|---|---|
| Anemia | Most patients referred for HSCT are anemic and transfusion dependent, not prognostic for posttransplant outcomes | Ferritin <1000 μg/L: • Careful transfusion support Ferritin >1000 μg/L: • Deferasirox (14 mg/kg) from the start of conditioning until day 3 after transplantation, and monitor ferritin closely until at least day 14 • Pharmacological monitoring for busulfan AUC Peritransplant transfusion threshold: • <8 g/dL if patients usually achieved >8 g/dL before transplant referral • <7 g/dL if patients never achieved >8 g/dL before transplant referral and are not symptomatic (clinically adapted to anemia) Momelotinib is an investigational option for anemic and progressive disease while preparing for HSCT |
| Thrombocytopenia | Associated with worse survival and nonrelapse mortality | Peritransplant transfusion threshold: • <10 × 109/L with no signs of bleeding • HLA-matched products in refractory cases and/or antiplatelet antibodies • Investigational eltrombopag or romiplostim in refractory cases, at high risk of and/or signs of bleeding Pacritinib is an investigational option for severe thrombocytopenia, and fedratinib for moderate thrombocytopenia and progressive disease while preparing for HSCT |
| Circulating blasts | Not associated with overall survival or nonrelapse mortality. Associated with increased risk of relapse in accelerated-phase MF. | No blast reduction in chronic-phase MF but close monitoring for relapse Venetoclax and/or azacitidine is an investigational option for accelerated and blast phase while preparing for HSCT but in need of treatment |
| Splenomegaly | Not associated with mortality. Associated with delayed engraftment, increased risk of graft failure, and relapse. | Start with JAK inhibitor (mostly ruxolitinib) and proceed directly to HSCT for eligible patients with spleen response or moderate or large spleen size with acceptable performance status and low or intermediate transplant-specific risk Fedratinib or investigational JAK inhibitors or clinical trials for patients with large spleen, cytopenia, and/or poor performance with high transplant-specific risk Splenic irradiation as part of HSCT preparation for patients with massive spleen, not responding to prior therapy, and no severe cytopenia Splenectomy only for very few selected patients with excellent performance |
| Condition . | Association with outcome . | Management . |
|---|---|---|
| Anemia | Most patients referred for HSCT are anemic and transfusion dependent, not prognostic for posttransplant outcomes | Ferritin <1000 μg/L: • Careful transfusion support Ferritin >1000 μg/L: • Deferasirox (14 mg/kg) from the start of conditioning until day 3 after transplantation, and monitor ferritin closely until at least day 14 • Pharmacological monitoring for busulfan AUC Peritransplant transfusion threshold: • <8 g/dL if patients usually achieved >8 g/dL before transplant referral • <7 g/dL if patients never achieved >8 g/dL before transplant referral and are not symptomatic (clinically adapted to anemia) Momelotinib is an investigational option for anemic and progressive disease while preparing for HSCT |
| Thrombocytopenia | Associated with worse survival and nonrelapse mortality | Peritransplant transfusion threshold: • <10 × 109/L with no signs of bleeding • HLA-matched products in refractory cases and/or antiplatelet antibodies • Investigational eltrombopag or romiplostim in refractory cases, at high risk of and/or signs of bleeding Pacritinib is an investigational option for severe thrombocytopenia, and fedratinib for moderate thrombocytopenia and progressive disease while preparing for HSCT |
| Circulating blasts | Not associated with overall survival or nonrelapse mortality. Associated with increased risk of relapse in accelerated-phase MF. | No blast reduction in chronic-phase MF but close monitoring for relapse Venetoclax and/or azacitidine is an investigational option for accelerated and blast phase while preparing for HSCT but in need of treatment |
| Splenomegaly | Not associated with mortality. Associated with delayed engraftment, increased risk of graft failure, and relapse. | Start with JAK inhibitor (mostly ruxolitinib) and proceed directly to HSCT for eligible patients with spleen response or moderate or large spleen size with acceptable performance status and low or intermediate transplant-specific risk Fedratinib or investigational JAK inhibitors or clinical trials for patients with large spleen, cytopenia, and/or poor performance with high transplant-specific risk Splenic irradiation as part of HSCT preparation for patients with massive spleen, not responding to prior therapy, and no severe cytopenia Splenectomy only for very few selected patients with excellent performance |
AUC, area under the curve.