Rare Disease

The current treatment landscape of polycycthemia vera (PV) hinges on treatments like hydroxyurea and ruxolitinib, the latter having emerged as an effective second-line therapy in patients with severe pruritis, symptomatic splenomegaly, or post-PV myelofibrosis symptoms.

Researchers discuss the unique fetal and maternal challenges for pregnant women with myeloproliferative neoplasms, with insight and recommendations provided on the potential benefit of aspirin therapy, cytoreductive therapy, and systemic anticoagulation.

According to the report, direct medical costs represented less than half ($418 billion, or 43%) of overall annual costs.

The review comes amid growing attention directed at identifying treatments that can prevent progression of polycythemia vera (PV) to myelofibrosis or an aggressive form of acute myeloid leukemia.

Ruben Mesa, MD, leads a discussion on key benchmarks and final thoughts about good-quality care programs for patients with MPNs, including parameters for judging efficacy and safety.

Jamile Shammo, MD, Ruben Mesa, MD, and Michael Reff, RPh, MBA, discuss having pharmacists on MPN care teams and the effect they have on patient compliance.

A new study finds no link between the UK COVID-19 variant and more severe disease; study finds more than 1 in 10 US children with COVID-19 hospitalized; therapy setbacks in Huntington disease.

The study also indicated that the type of transplantation donor impacted outcomes following the procedure.

Michael Reff, RPh, MBA, provides insight on adherence programs for MPNs and discusses why patients may not be adherent to therapies.

Kathy Oubre and Michael Reff, RPh, MBA, address the lack of treatment adherence among patients with MPNs and how to overcome this with a best-practice approach.

According to authors, magnetic resonance imaging (MRI) should be used when incomplete signs of the rare complication are presented to help guide the skin biopsy.

According to a recent review, data suggest mast cells have an important role in tissue homeostasis and wound healing, with mass cell dysregulation potentially leading to fibrotic disease.

Ruben Mesa, MD, shares his thoughts on stem cell transplantation related to progression-free survival in MPNs, and Kathy Oubre highlights the importance of good working relationships.

Jamile Shammo, MD, discusses creating individual treatment plans for MPNs and following patients to have a better understanding of how they will respond to treatment.

Although the FDA has approved 599 orphan drugs since 1983, patent laws and other exclusivities keep most from any competition.

A Swedish study analyzed 5 years’ worth of data from over 3000 patients, 40% of whom received a molecular diagnosis.

Michael Reff, RPh, MBA; Kathy Oubre; and Ruben Mesa, MD, discuss how telehealth, technology, and innovation have increased patient adherence and created touch points with patients with MPNs.

Jamile M. Shammo, MD, comments on telehealth and the impact on patients who have MPNs during the COVID-19 pandemic.

According to the researchers, few treatments have been able to reduce the thrombotic burden in patients with polycythemia vera (PV) and essential thrombocytopenia (ET), and progress on this front has been hindered by a lack of studies designed to assess a treatment’s impact on thrombotic events.

The researchers of the study say their findings may aid the hematological community in determining the value of integrating gene expression profiles in contemporary prognostic models to better identify high-risk patients with a poor prognosis.

Kathy Oubre and Michael Reff, RPh, MBA, reflect on reactions to new guidelines for managing MPNs and how practices are integrating the new process, while Ruben Mesa, MD, shares his experience.

Ruben Mesa, MD, and Jamile M. Shammo, MD, discuss treatment guidelines, pathways, and pharmacologic treatment options that play a role in the standard of care for MPNs.

While the findings of the small study indicate that there was no statistically significant difference in the rate of vascular complications between White and non-White patients with disease, there were differences in survival.

Study findings detail the researchers’ ability to prevent stroke in 11 patients ranging in age from 18 months to 15 years

Michael Reff, RPh, MBA, and Kathy Oubre explain the internal guiding documents that aid a practice’s decision-making and workflow when treating patients with MPNs.