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Development and Validation of an Ultrasensitive Single Molecule Array Digital Enzyme-linked Immunosorbent Assay for Human Interferon-α
Published on: June 14, 2018
Recombinant interferon-α may retard progression of early primary myelofibrosis: a preliminary report
Richard T Silver1, Katherine Vandris, Joshua J Goldman
1Division of Hematology and Medical Oncology, Department of Medicine, Weill Cornell Medical College, 1305 York Avenue, New York, NY 10021, USA. rtsilve@med.cornell.edu
Abstract:
The limited effects of current treatments of primary myelofibrosis (PM) led us to prospectively evaluate recombinant interferon-α (rIFNα) in "early" PM patients with residual hematopoiesis and only grade 1 or 2 myelofibrosis. Seventeen patients meeting World Health Organization PM diagnostic criteria received either rIFNα-2b 500 000 to 3 million units 3 times weekly, or pegylated rIFNα-2a 45 or 90 μg weekly. International Working Group for Myelofibrosis Research and Treatment criteria for prognosis and response were used. Eleven patients were women and 6 were men. Their median age at diagnosis was 57 years. Eleven patients were low risk and 6 were intermediate-1 risk. Two achieved complete remission, 7 partial, 1 clinical improvement, 4 stable disease, and 3 had progressive disease. Thus, more than 80% derived clinical benefit or stability. Improvement in marrow morphology occurred in 4. Toxicity was acceptable. These results, with documented marrow reversion because of interferon treatment, warrant expanded evaluation.
Insights
Recombinant interferon-alfa (rIFNα) shows promise for early primary myelofibrosis patients. Over 80% experienced clinical benefit or stability, with some marrow reversion, suggesting rIFNα as a viable treatment option.
Area of Science:
- Hematology
- Oncology
- Pharmacology
Background:
- Primary myelofibrosis (PM) has limited effective treatments.
- Early-stage PM patients with residual hematopoiesis and low-grade fibrosis require novel therapeutic approaches.
Purpose of the Study:
- To prospectively evaluate the efficacy and safety of recombinant interferon-alfa (rIFNα) in early primary myelofibrosis patients.
- To assess treatment response using International Working Group for Myelofibrosis Research and Treatment criteria.
Main Methods:
- Seventeen "early" PM patients received either rIFNα-2b or pegylated rIFNα-2a.
- Treatment involved doses ranging from 500,000 to 3 million units thrice weekly or 45-90 μg weekly.
- Patient prognosis and response were monitored.
Main Results:
- Over 80% of patients (14/17) achieved clinical benefit or disease stability.
- Two patients achieved complete remission, and seven achieved partial remission.
- Four patients had stable disease, and four showed improvement in marrow morphology.
Conclusions:
- Recombinant interferon-alfa demonstrates significant clinical benefit and stability in early primary myelofibrosis.
- The treatment is associated with acceptable toxicity and documented marrow reversion.
- Expanded evaluation of rIFNα for early PM is warranted.
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