Myelofibrosis Drug Summary

Last updated: 14 March 2025

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Cytotoxic Chemotherapy

Content on this page:

Cytotoxic Chemotherapy

Cytotoxic Chemotherapy



Drug Dosage Remarks
Azacitidine Initial dose: 75 mg/m2 SC/IV infusion over 10-40 minutes on days 1-7 of a 28-day treatment cycle
May increase to 100 mg/m2 if no benefit after 2 cycles and no toxicity
Continue until disease progression		 Adverse Reactions
  • GI effects (nausea/vomiting, abdominal pain, diarrhea, anorexia, constipation, dyspepsia, mucositis); Hematologic effects (anemia, neutropenia, thrombocytopenia, GI or intracranial hemorrhage, bruising, petechiae); CNS effects (headache, insomnia, dizziness, anxiety); Dermatologic effects (alopecia, itch, rash); Other effects (renal tubular acidosis, elevated serum creatinine, injection site reactions hepatotoxicity, dyspnea, hypokalemia, fever, infection)
Special Instructions
  • Contraindicated in patients with advanced malignant hepatic tumor
  • Use with caution in patients with high tumor burden, history of severe chronic heart failure, clinically unstable cardiac disease or pulmonary disease, renal and severe hepatic impairment
  • Monitor CBC, LFTs, electrolytes and renal function at baseline and before each treatment cycle
Busulfan 2-4 mg PO 24 hourly Adverse Reactions
  • Dose-related bone marrow depression; GI effects (nausea/vomiting, diarrhea, oral ulceration); Hepatic effects (jaundice, hyperbilirubinemia, hepatic veno-occlusive disease); Dermatologic effects (alopecia, hyperpigmentation); Other effects (ovarian suppression and amenorrhea in females, sterility and testicular atrophy in males)
Special Instructions
  • Take with chilled liquid
  • Ensure adequate fluid intake
  • Premedication with prophylactic anticonvulsants and antiemetics is recommended if high-dose treatment for HSCT is prescribed
  • Use with caution in patients with compromised bone marrow reserve due to previous radiation therapy, prior chemotherapy or prior stem cell transplantation, history of seizures or taking antiepileptic agents, history of head trauma, renal and hepatic impairment
  • Avoid concomitant administration of live vaccines
  • Monitor CBC during treatment
Decitabine 20 mg/m2 IV infusion over 60 minutes for 5 consecutive days
Repeat cycle every 4 weeks for a minimum of 4 cycles 		Adverse Reactions
  • Hematologic effects (neutropenia, thrombocytopenia, anemia, leukopenia); GI effects (nausea/vomiting, diarrhea, stomatitis); Other effects (pneumonia, UTI, other infections, pyrexia, septic shock, sepsis, sinusitis, epistaxis)
Special Instructions
  • Use with caution in patients with history of severe chronic heart failure or clinically unstable cardiac disease, hepatic and severe renal impairment
  • Monitor CBC and platelet count at baseline and before each treatment cycle
  • Monitor for signs and symptoms of infection and treat promptly
Hydroxyurea
(Hydroxycarbamide)		 20-30 mg/kg PO as single dose 24 hourly or 80 mg/kg PO single dose 72 hourly
Concomitant therapy: 80 mg/kg PO single dose 72 hourly, started at least 7 days before initiation of radiotherapy		 Adverse Reactions
  • GI effects (nausea/vomiting, diarrhea, anorexia); Other effects (decreased blood counts, alopecia, headache)
Special Instructions
  • May be taken with or without food
  • Contraindicated in patients with hypersensitivity to Hydroxyurea, marked bone marrow depression
  • Use with caution in the elderly
  • Monitor blood counts

Targeted Cancer Therapy



Drug Dosage Remarks
Fedratinib Baseline platelet count ≥50,000/mm3:
400 mg PO 24 hourly
Reduce or modify dose in the presence of adverse reactions and in patients who become transfusion dependent during therapy 		Adverse Reactions
  • CNS effect (fatigue); Metabolic effects (increased amylase, hyponatremia, increased serum lipase); GI effects (nausea/vomiting, diarrhea); Hematologic effects (anemia, thrombocytopenia, neutropenia); Hepatic effects (increased AST and ALT); Other effects (increased serum creatinine, asthenia, muscle spasm)
Special Instructions
  • Administration with high-fat meal may reduce the incidence of nausea/vomiting
  • Avoid use in patients with severe hepatic impairment
  • Adjust dose in patients with severe renal impairment
  • Perform CBC at baseline and periodically during treatment
  • Assess thiamine level prior to initiation and periodically during course of treatment
    • Replete thiamine before initiation of treatment if with thiamine deficiency
    • Discontinue temporarily if encephalopathy is suspected (signs and symptoms include ataxia, mental status changes, nystagmus, diplopia) then initiate parenteral Thiamine and monitor until symptoms resolve and thiamine levels normalize
  • Monitor hepatic function, amylase and lipase at baseline and periodically during treatment
  • Discontinue if unable to tolerate 200 mg/day dose
Momelotinib 200 mg PO 24 hourly until disease progression or unacceptable toxicity
Adverse Reactions
  • Hematologic effects (hemorrhage, thrombocytopenia); GI effects (abdominal pain, diarrhea, nausea); CNS effects (dizziness, fatigue, headache); Hepatic effects (increased serum AST, ALT, bilirubin); CV effects (hypotension, peripheral edema); Dermatologic effects (pruritus, skin rash); Other effects (cough, fever, infection, limb pain)
Special Instructions
  • Avoid use in patients with active infection
  • Adjust dose in patients with severe hepatic impairment
  • Monitor for signs and symptoms of infection including reactivation of hepatitis B, cardiovascular events, thrombosis and development of secondary malignancies; perform CBC with platelet count and LFTs at baseline and periodically during treatment
Pacritinib Platelet count <50 x 109/L:
200 mg PO 12 hourly		 Adverse Reactions
  • Hematologic effects (anemia, thrombocytopenia); GI effects (diarrhea, nausea); Other effect (peripheral edema)
Special Instructions
  • Avoid use in patients with active bleeding, baseline QTc >480 msec
  • Use with caution in patients with diarrhea, thrombocytopenia, major adverse cardiac events, thrombosis, increased risk for secondary malignancies or infection
  • Contraindicated in patients taking strong CYP3A4 inhibitors or inducers
Ruxolitinib Initial dose:
Platelet count 50,000-<100,000/mm3:
5 mg PO 12 hourly
Platelet count 100,000-200,000/mm3:
15 mg PO 12 hourly
Platelet count >200,000/mm3:
20 mg PO 12 hourly
Titrate dose based on efficacy and tolerability after 4 weeks then at 2-week intervals in
5-mg increments 12 hourly
Max dose: 25 mg PO 12 hourly 		Adverse Reactions
  • Hematologic effects (neutropenia, anemia, thrombocytopenia); CNS effects (headache, dizziness, progressive multifocal leukoencephalopathy); Other effects (increased AST and ALT, bruising, hypercholesterolemia, non-melanoma skin cancers)
Special Instructions
  • Interrupt dosing if platelet count <50,000/mm3 or absolute neutrophil count (ANC) <500/mm3
  • Contraindicated in patients with hypersensitivity to Ruxolitinib
  • Use with caution in patients with hepatic and moderate to severe renal impairment, at risk of hematological adverse reactions (thrombocytopenia, anemia, neutropenia), with TB or at risk for TB and other serious bacterial, fungal and viral infections, chronic hepatitis B infection and in patients taking CYP3A4 inhibitors (eg Fluconazole)
  • Perform CBC with differential at baseline and regularly during the course of treatment; periodic lipid monitoring, renal and hepatic function tests, and skin examination during the course of treatment
  • Patient should be advised about signs and symptoms of herpes zoster and must seek treatment as early as possible
  • Discontinue if without improvement of symptoms or reduction in spleen size after 6 months
  • Avoid sudden withdrawal; taper gradually if discontinuing for reasons other than thrombocytopenia or neutropenia

Androgens & Related Synthetic Drugs

Drugs for Myelofibrosis-Associated Anemia

Drug Dosage Remarks
Danazol Initial dose: 200 mg PO 12 hourly
May increase to 400 mg PO 12 hourly as tolerated
Continue for minimum of 3 months and dose may be tapered to minimum effective dose after 6 months

Adverse Reactions
  • Androgenic effects (acne, seborrhea, weight gain, hair loss, voice change, clitoral hypertrophy); Hepatic effects (hepatitis, benign hepatic adenoma, cholestatic jaundice); CNS effects (nervousness, emotional lability, headache); CV effects (palpitations, tachycardia, benign intracranial hypertension); Other effects (interstitial pneumonitis, menstrual disturbances)
Special Instructions
  • Contraindicated in patients with thromboembolic disease or history of thrombosis, severe cardiac impairment, severe hepatic or renal impairment, undiagnosed abnormal genital bleeding, androgen-dependent tumor
  • Use with caution in patients with epilepsy, migraine, DM, polycythemia, hypertension and other CV disease, lipoprotein disorder, hepatic or renal impairment
  • Monitor LFTs at baseline and regularly during treatment
  • Screen men for prostate cancer before initiation of therapy and during treatment
  • Monitor for signs and symptoms of intracranial hypertension, androgenic changes and/or fluid retention
  • Perform CBC, lipid panel, renal function tests regularly

Cancer Immunotherapy

Drugs for Myelofibrosis-Associated Anemia

Drug Dosage Remarks
Lenalidomide 10 mg PO 24 hourly on days 1-21 of repeated 28-day cycles

Adverse Reactions
  • Hematologic effects (thrombocytopenia, neutropenia, leukopenia, anemia); Respiratory effects (pneumonia, lung infection, bronchitis, nasopharyngitis, cough); CV effects (venous thromboembolism, pulmonary embolism, hypotension, peripheral edema); GI effects (diarrhea, nausea, decreased appetite); CNS effects (insomnia, asthenia, peripheral neuropathy); Other effects (renal failure, muscle spasms, pyrexia, fatigue, back pain, hypocalcemia, pruritus)
Special Instructions
  • Avoid use in patients with high tumor burden
  • Use with caution in patients with known risk factors for thromboembolism or MI, hepatic or renal impairment, concomitant use with erythropoietic agents
  • Monitor CBC with differential count, platelet count every week for the first 8 weeks of treatment then every month thereafter
  • Monitor LFTs regularly
Thalidomide 50-200 mg PO 24 hourly

Adverse Reactions
  • CNS effects (severe and irreversible peripheral neuropathy, dizziness, drowsiness, somnolence); CV effects (bradycardia, orthostatic hypotension); Other effects (constipation, hypersensitivity reaction)
Special Instructions
  • Should be taken on an empty stomach or at least 1 hour after a meal
  • Use with caution in patients with high tumor burden
  • Monitor CBC with differential count regularly

Haematopoietic Agents

Drugs for Myelofibrosis-Associated Anemia

Drug Dosage Remarks
Darbepoetin alfa 150-300 mcg SC weekly
or
 500 mcg SC once every 2-3 weeks 		Adverse Reactions
  • CV effects (hyper-/hypotension, chest pain, peripheral edema); CNS effects (dizziness, headache); Musculoskeletal effects (myalgia, arthralgia, limb pain); Respiratory effects (cough, dyspnea, bronchitis); Other effects (fatigue, fever, skin reactions, hyperkalemia, infection, transient increase in platelet count, influenza-like symptoms, injection site pain)
Special Instructions
  • Contraindicated in patients with uncontrolled hypertension
  • Use with caution in patients with hypertension, history of seizures, hepatic impairment, CV disease, thrombocytosis
  • Monitor BP, hemoglobin, electrolytes and platelet counts regularly
Epoetin alfa 10,000-20,000 units SC 3 times weekly

Other Antianemic Preparation

Drugs for Myelofibrosis-Associated Anemia

Drug Dosage Remarks
Luspatercept 1 mg/kg SC 3 weekly

Adverse Reactions
  • GI effects (nausea, diarrhea, abdominal pain); Musculoskeletal effects (musculoskeletal pain, arthralgia); Respiratory effects (cough, dyspnea); CNS effects (headache, dizziness/vertigo); Other effects (fatigue, hypersensitivity)
Special Instructions
  • Use with caution in patients with beta thalassemia
  • Monitor BP, for signs and symptoms of thromboembolic events