Table 2.
Conventional drug treatment strategies in myelofibrosis.
| Indications | Treatment options | Reported response rates | Side effects include |
|---|---|---|---|
| Anemia | Thalidomide 50 mg QD + Prednisone 20 mg QD (171, 172) | 46%–62% | Peripheral neuropathy |
| Constipation | |||
| Fatigue | |||
| Cutaneous reactions | |||
| CNS symptoms/sedation | |||
| Sleep disturbances | |||
| Anabolic steroids (173, 174) (dose depends on preparation) | 44%–57% | Virilizing effects | |
| Liver toxicity | |||
| Danazol 200 mg BID (175) | 30% (175) | Liver toxicity | |
| LFT abnormalities | |||
| Lenalidomide 10 mg QD ± Prednisone 20 mg QD (176, 177) | 20%–25% | Myelosuppression | |
| ESAs (114, 178) | 0% in Tx-dependent (114) | Unremarkable | |
| 37% in Tx-independent (114) | |||
| Splenomegaly | Hydroxyurea 500 mg BID (112) (starting dose) | 40%b | ↓hemoglobin; ↓platelet count |
| Skin changes including ulcers | |||
| Oral ulcers | |||
| Nail discolorations | |||
| Ruxolitinib 15 mg BID (117, 120) (starting dose) | 32%a | ↓hemoglobin; ↓platelet count | |
| Herpes zoster (180) | |||
| Reactivation of tuberculosis (180) | |||
| Other opportunistic infections (180) | |||
| ↓COVID-19 vaccine response (126) | |||
| Fedratinib 400 mg QD (129, 179) | 47%a | ↓hemoglobin; ↓platelet count | |
| ↑LFTs; ↑pancreatic enzymes | |||
| Gastrointestinal symptoms | |||
| Encephalopathyb | |||
| Constitutional symptoms | Ruxolitinib 15 mg BID (117, 120) | Majority of patients | See above |
| Fedratinib 400 mg QD (129, 179) | Majority of patients | See above |
Abbreviations: BID, twice-daily; ESAs, erythropoiesis-stimulating agents including darbepoetin 150–300 g every 2 weeks; LFT, liver function test; Tx, red blood cell transfusion; QD, once-daily.
aSeen at 500 mg/day dose.
bResponse rates are not comparable because of reference to different patient populations and different methods and timing of spleen response assessment.