Medical therapy for symptomatic splenomegaly in myelofibrosis (available medications)
| Agent . | Administration route . | Dose/schedule . | Response rate* . | Additional efficacy . | Toxicities . | Reference . |
|---|---|---|---|---|---|---|
| Tier 1 | ||||||
| Hydroxyurea | Oral | 500-3000 mg daily | 40%-50% | None | Myelosuppression, skin ulcers | 10 |
| Busulfan | Oral | 2-4 mg daily | Variable | None | Myelosuppression, leukemia | 11 |
| Melphalan | Oral | 2.5 mg 3 times weekly | 67% | None | Myelosuppression, leukemia | 12 |
| Interferon-2α | Subcutaneous | 0.5-1.0 × 106 units 3 times weekly | Limited data; 75% with small spleens, less with larger | None | Myelosuppression, depression | 13 |
| Thalidomide with or without prednisone taper | Oral | 50 mg daily | 19% | Anemia, thrombocytopenia | Neuropathy, sedation, myeloproliferation | 14 |
| Lenalidomide with or without prednisone taper | Oral | 5-10 mg daily | 33% | Anemia, thrombocytopenia | Myelosuppression, rash, diarrhea | 15 |
| Tier 2 | ||||||
| Cladribine (2-CdA) | Intravenous | 5 mg/m2 per day × 5 days per month | 56% | None | Myelosuppression | 16 |
| Daunorubicin | Intravenous | 60 mg/m2 on days 1-3 | NA | None | Myelosuppression, cardiotoxicity | None |
| 5-Azacytidine | Subcutaneous | 75 mg/m2 on days 1-7 per month | 21% | Anemia | Myelosuppression, gastrointestinal | 17,18 |
| Decitabine | Subcutaneous | 20 mg/m2 on days 1-5 per month | NA | Myelosuppression | None |
| Agent . | Administration route . | Dose/schedule . | Response rate* . | Additional efficacy . | Toxicities . | Reference . |
|---|---|---|---|---|---|---|
| Tier 1 | ||||||
| Hydroxyurea | Oral | 500-3000 mg daily | 40%-50% | None | Myelosuppression, skin ulcers | 10 |
| Busulfan | Oral | 2-4 mg daily | Variable | None | Myelosuppression, leukemia | 11 |
| Melphalan | Oral | 2.5 mg 3 times weekly | 67% | None | Myelosuppression, leukemia | 12 |
| Interferon-2α | Subcutaneous | 0.5-1.0 × 106 units 3 times weekly | Limited data; 75% with small spleens, less with larger | None | Myelosuppression, depression | 13 |
| Thalidomide with or without prednisone taper | Oral | 50 mg daily | 19% | Anemia, thrombocytopenia | Neuropathy, sedation, myeloproliferation | 14 |
| Lenalidomide with or without prednisone taper | Oral | 5-10 mg daily | 33% | Anemia, thrombocytopenia | Myelosuppression, rash, diarrhea | 15 |
| Tier 2 | ||||||
| Cladribine (2-CdA) | Intravenous | 5 mg/m2 per day × 5 days per month | 56% | None | Myelosuppression | 16 |
| Daunorubicin | Intravenous | 60 mg/m2 on days 1-3 | NA | None | Myelosuppression, cardiotoxicity | None |
| 5-Azacytidine | Subcutaneous | 75 mg/m2 on days 1-7 per month | 21% | Anemia | Myelosuppression, gastrointestinal | 17,18 |
| Decitabine | Subcutaneous | 20 mg/m2 on days 1-5 per month | NA | Myelosuppression | None |
NA indicates not applicable.