Expert Panel Discusses Advocacy, Education for Patients With Myeloproliferative Neoplasms


Despite a slew of barriers with patient management, pharmacists have the training and resources to advocate for better patient care and help patients with hematologic myeloproliferative neoplasms advocate for themselves.

Patients with hematologic myeloproliferative neoplasms (MPNs)—a group of rare blood diseases that include myelofibrosis, essential thrombocythemia (ET), and polycythemia vera (PV)—should take a more active role in their treatment plan, according to experts in oncology pharmacy who participated in a Pharmacy Times clinical forum at ASCO 2023 in Chicago, Illinois.

Credit: MdBabul -

Credit: MdBabul -

“I think that our role as pharmacists is to give [patients] as much information as we possibly can, and then encourage them to move forward with advocating for themselves,” said Krystal Preston, PharmD, BCPS, senior clinical specialist-oncology pharmacist, CVS Health, and professor, University of Chicago College of Pharmacy, Chicago, Illinois.

Patients who are serious about taking on an active role in their treatment could inspire health care providers to collaborate more both with them and other members of the care team, according to discussion leader Zahra Mahmoudjafari, PharmD, MBA, BCOP, FHOPA, clinical pharmacy manager of hematology, bone marrow therapy, and cellular therapeutics at the University of Kansas Health System in Mission, Kansas.

Myelofibrosis, ET, and PV are subtypes of MPNs. ET and PV typically transform into myelofibrosis, which can subsequently turn into acute myeloid leukemia (AML). At least 20% of MPNs may transform into AML, therefore, the goal for treatment is to prevent this from occurring, Mahmoudjafari explained.

More than 90% of patients with PV have a Janus kinase 2 gene (JAK2) mutation, “and it is probably, by far, the mutation that we have the most actionable ability to do something about,” Mahmoudjafari said. She added that because there are currently 3 FDA-approved JAK inhibitors for MPN—ruxolitinib (Opzelura; Incyte), fedratinib (Inrebic; Bristol Myers Squibb), and pacritinib (Vonjo; CTI Biopharma Corp.), which were approved based on results from the COMFORT-1, JAKARTA, and PERSIST-2 pivotal trials, respectively.

Ruxolitinib and fedratinib are primarily for patients with intermediate- or high-risk myelofibrosis, including intermediate-2, and primary and post-PV/ET myelofibrosis. Pacritinib is indicated for patients with a platelet count below 50,000, and all have an expected adverse events profile, which includes thrombocytopenia, anemia, bruising, dizziness, headache, and diarrhea.

The only true cure for myelofibrosis is transplant, however, there is a 30% mortality risk associated with it, Mahmoudjafari said. During the forum, panelists largely explored patient management. Compliance was cited as a predominant issue for Connor Roth, PharmD, BCOP, hematology/oncology pharmacy specialist, Franciscan Alliance, Inc, Chicago, Illinois. Whether it’s due to dosing schedule, toxicities, cost, or all of the above, “people are just forgetful,” Roth said.

Currently, it’s much harder to contact patients with a reminder via phone call because, “nobody picks up a phone number they don't know,” Roth added.

Tammy McClellan, PharmD, a clinical oncology pharmacist at Riverside Healthcare in Kankakee, Illinois, said that one of the greatest unmet needs she is seeing is timely access to medications. Timely access is critical because the faster a patient can get on a proper treatment regimen, the better they can prevent a blood-clotting event, according to the panelists.

Insurance is a barrier to access; however, pharmacists understand how to work within the system and are best positioned to advocate for patients, Roth said. Location is equally important for access to medications because patients living close to a city can access treatment centers and pharmacies more easily than those in a rural setting. Patients in cities also have better access to clinical trials, Preston said.

McClellan noted that an unmet patient need is effective communication with their care providers. She said that patients frequently complain that they wish their provider would listen to their input more often.

The clinical oncology pharmacist said a solution to this problem may be individualized patient care. Pharmacists and providers can foster individualized care through better organized collaboration with the patient and care team, Radhakrishnan said. This can make it easier to manage AEs and drug-drug interactions because treatment is exceedingly difficult, according to Mahmoudjafari. Therefore, improving AE management can improve patient quality of life.

“[Symptoms can be] enough to drive these patients absolutely insane,” McClellan added.

Financial burden is a significant issue for many patients, therefore, some clinics have financial navigators who work with pharmacists and patients to coordinate benefits, co-pays, and prior authorization. Other institutions may assign these tasks to specialty pharmacists, who typically have experience with patient assistance programs, which help older adults or people with limited resources to access affordable medications via grants, foundational support, or other means. Ideally, insurance or patient assistance would be connected to the patient’s electronic medical record, according to Roth. The panelists also made sure to emphasize patient education.

“I really try to explain to [patients], in layman's terms, what's going on and just kind of listen to what their issues are and what their concerns are,” Preston said.

The panelists said that a best practice is to provide as much information about the disease state and treatment as possible. Many patients do not understand their disease state, therefore, improving their understanding can provide the patient with more control, help them learn how to express their concerns, and to be their own advocate.

“You can't make the assumption that the patient already knows [everything],” Mahmoudjafari said.

This is especially important because oncologists or other providers may be struggling to keep up with a complicated and shifting treatment and guidelines landscape.

“Guidelines are dividing, and the drugs are—there's so many things to know,” Roth said. “Pharmacists can be the ones to extend the hands of the physicians and be a patient advocate when [the patient] doesn’t always have one.”


American Society for Clinical Oncology. Pharmacy Times clinical forum. ASCO Annual Meeting 2023. June 2 to 6, 2023. Chicago, Illinois. Accessed July 13, 2023.

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