Supportive care for cytopenic MF
| Drug class . | Indication . | Agent . | Key data . | Toxicities of note . | References . |
|---|---|---|---|---|---|
| ESAs | Anemia, if endogenous Epo level <500 mU/mL | Darbopoetin alpha Epoetin alpha | Retrospective, multicenter study of patients with MF and anemia: - Anemia response in 53%. - Median duration of response 19 mo. Retrospective study in the setting of concurrent Rux treatment showed anemia response in 54%. | - Very few toxicities but hypertension and thromboses are possible. | 59,60 |
| TGF-β inhibitors | Anemia | Luspatercept | Phase 2 study of patients with MF and anemia: - On luspatercept monotherapy, 10% converted from transfusion dependent to independent. - On luspatercept plus Rux, 27% converted to transfusion independence. - Similar response rates among transfusion-independent groups. Ongoing phase 3 study of luspatercept vs placebo in patients with MF on Jak inhibitor therapy who require transfusions (NCT04717414). | - Very few toxicities but hypertension and bone pain are possible. | 48 |
| Sotatercept | Phase 2 study showed anemia responses in 30% with sotatercept monotherapy and 32% with combination therapy with Rux. | 49 | |||
| IMiDs | Anemia, thrombocytopenia | Thalidomide | Phase 2 study in 63 patients with MF, increase in platelet count by >50 × 109/L in 22% with baseline thrombocytopenia. Pooled analysis of MF studies using thalidomide at doses ≥100 mg/d; 38% had an increase in platelet counts, and 29% had an increase in Hb. | - Constipation - Fatigue - Peripheral neuropathy - Neutropenia - Sedation - Edema | 61,62 |
| Lenalidomide | Pooled data from MF studies showed that 50% of patients had a platelet response (>50% improvement from baseline to absolute count of >50 × 109/L). | 63 | |||
| Pomalidomide | Phase 3 study showed platelet response in 22% on Pom vs 0% on placebo. | 64 | |||
| Androgen | Anemia, thrombocytopenia | Danazol | In 50 patients with MF and anemia: - WHO IWG-defined anemia response achieved in 15 (30%), including 5/27 (18%) of those with transfusion dependency. - Median time to response 5 mo - Median duration of response 14 mo - Among 13 patients with thrombocytopenia, a platelet response was seen in 3 (23%). | - Hepatic toxicity - Prostate cancer risk | 65 |
| TPO receptor agonists | Thrombocytopenia (not routinely recommended) | Eltrombopag | Among 6 patients with MF on Rux with thrombocytopenia, 0 had a sustained platelet response. | - Has not been effective - Theoretic risk of worsening fibrosis - Not routinely recommended | 66 |
| Drug class . | Indication . | Agent . | Key data . | Toxicities of note . | References . |
|---|---|---|---|---|---|
| ESAs | Anemia, if endogenous Epo level <500 mU/mL | Darbopoetin alpha Epoetin alpha | Retrospective, multicenter study of patients with MF and anemia: - Anemia response in 53%. - Median duration of response 19 mo. Retrospective study in the setting of concurrent Rux treatment showed anemia response in 54%. | - Very few toxicities but hypertension and thromboses are possible. | 59,60 |
| TGF-β inhibitors | Anemia | Luspatercept | Phase 2 study of patients with MF and anemia: - On luspatercept monotherapy, 10% converted from transfusion dependent to independent. - On luspatercept plus Rux, 27% converted to transfusion independence. - Similar response rates among transfusion-independent groups. Ongoing phase 3 study of luspatercept vs placebo in patients with MF on Jak inhibitor therapy who require transfusions (NCT04717414). | - Very few toxicities but hypertension and bone pain are possible. | 48 |
| Sotatercept | Phase 2 study showed anemia responses in 30% with sotatercept monotherapy and 32% with combination therapy with Rux. | 49 | |||
| IMiDs | Anemia, thrombocytopenia | Thalidomide | Phase 2 study in 63 patients with MF, increase in platelet count by >50 × 109/L in 22% with baseline thrombocytopenia. Pooled analysis of MF studies using thalidomide at doses ≥100 mg/d; 38% had an increase in platelet counts, and 29% had an increase in Hb. | - Constipation - Fatigue - Peripheral neuropathy - Neutropenia - Sedation - Edema | 61,62 |
| Lenalidomide | Pooled data from MF studies showed that 50% of patients had a platelet response (>50% improvement from baseline to absolute count of >50 × 109/L). | 63 | |||
| Pomalidomide | Phase 3 study showed platelet response in 22% on Pom vs 0% on placebo. | 64 | |||
| Androgen | Anemia, thrombocytopenia | Danazol | In 50 patients with MF and anemia: - WHO IWG-defined anemia response achieved in 15 (30%), including 5/27 (18%) of those with transfusion dependency. - Median time to response 5 mo - Median duration of response 14 mo - Among 13 patients with thrombocytopenia, a platelet response was seen in 3 (23%). | - Hepatic toxicity - Prostate cancer risk | 65 |
| TPO receptor agonists | Thrombocytopenia (not routinely recommended) | Eltrombopag | Among 6 patients with MF on Rux with thrombocytopenia, 0 had a sustained platelet response. | - Has not been effective - Theoretic risk of worsening fibrosis - Not routinely recommended | 66 |
Epo, erythropoietin; ESA, erythropoiesis stimulating agent; Hb, hemoglobin; Pom, pomalidomide; Rux, ruxolitinib; TPO, thrombopoietin; WHO IWG, World Health Organization International Working Group.