Dr John Mascarenhas (Icahn School of Medicine at Mount Sinai, New York, NY, USA) discusses pacritinib in the potential treatment of myelofibrosis and severe thrombocytopenia. Treatment options for myelofibrosis and severe thrombocytopenia are restricted due to high occurrences of JAK inhibitor-related thrombocytopenia.
The abstract entitled ‘PACIFICA: A Randomized, Controlled Phase 3 Study of Pacritinib Versus Physician’s Choice in Patients with Primary or Secondary Myelofibrosis and Severe Thrombocytopenia’ (Abstract #4316) was presented at ASH Annual Meeting, 10–13, December, 2022.
Questions:
- What are the challenges in the treatment of patients with myelofibrosis? (0:16)
- What is the rationale for the use of pacritinib in these patients? (0:44)
Disclosures: John Mascarenhas is a consultant for Abbvie, BMS, CTi Bio, Galecto, GSK, Incyte Roche, Kartos, Karyopharm, Morphosys, Novartis and PharmaEssentia. He has received grant/research support from Abbvie, BMS, CTI, Incyte, Kartos, Novartis and PharmaEssentia.
Support: Interview and filming supported by Touch Medical Media. Interview conducted by Danielle Crosby originally for touchONCOLOGY.com
Transcript:
My name is John Mascarenhas. I am a professor of Medicine at the Icahn School of Medicine at Mount Sinai in New York City.
What are the challenges in the treatment of patients with myelofibrosis? (0:16)
There are many challenges to treating patients with myelofibrosis as it is a complex disease at the biologic level and at the clinical level patients present in varied ways and have a heterogeneous clinical course. Their needs could be different. So really the approach and treatment to myelofibrosis has to be personalized and the treatment goals have to be recognized and shared between the patient and the physician.
What is the rationale for the use of pacritinib in these patients? (0:44)
So as we talk about the challenges of treating patients with myelofibrosis, one of the biggest challenges that can present is that of the site of myelofibrosis patient those patients that have low blood counts typified by usually extreme thrombocytopenia, frequently with anaemia, transfusion dependence. These patients can be quite symptomatic and they have a complex disease. They can be JAK-2 mutated. Often that burden is low. They are frequently harboring non-driver JAK mutations such as epigenetic alterations. So the biology is difficult and there is a significant aspect of bone marrow failure. The bone marrow is fibrotic, it is not capable of producing normal hematopoesis as it once did, and that really limits the ability to give a lot of available therapies, particularly JAK inhibitors that are often associated with treatment. Emergent side option is. So the clinic offers for the first time really an opportunity to provide spleen and symptom benefit and the potential for anemia benefit in patients with this cytopenic profile and it’s again marked by thrombocytopenia. So the label here in the US is for patients with myelofibrosis, irrespective of line of therapy and a platelet count of less than $50,000.
