July 11, 2012 — Seven hematologists from leading cancer centers have volunteered to provide free medical advice to patients with myeloproliferative neoplasms (MPNs), a rare form of leukemia, and to their healthcare providers.
The physicians have signed on with the online MPNforum Magazine, which is published monthly by an MPN patient collective. They offer help to patients and caregivers on MPN Clinic, an online roundtable hosted by the publication.
The term MPN covers a rare set of disorders occurring in about 5 people per million, according to a press release from the publication, which operates on a shoestring budget. This rarity means that community-based clinicians may never see a case and if one comes along, help is needed.
The initial volunteers for the project are Richard Silver, MD (Weill-Cornell in New York City), Srdan Verstovsek, MD (M.D. Anderson in Houston, Texas), Ruben Mesa, MD (Mayo Clinic in Scottsdale, Arizona), Claire Harrison, DM (Guy’s and St. Thomas’ in London, United Kingdom), Jason Gotlib, MD (Stanford Medical Center in Palo Alto, California), Ross Levine, MD (Sloan-Kettering in New York City), and Attilio Orazi, MD (Weill-Cornell).
“It’s amazing that we have this group of doctors,” said Zhenya Senyak, the founder and editor of MPNforum, about the eminence of the participating experts. “They are really concerned about getting this information out,” he told Medscape Medical News in an interview. Senyak, who lives in Asheville, North Carolina, is a writer who has myelofibrosis.
I’ve never waited more than a day for a response.
The service is free. Patients, caregivers, and healthcare providers can email questions to MPNclinic@gmail.com, which are then forwarded to all members of the panel. One of the experts responds, and circulates that response among the other panel members for review and comment. The final response is then forwarded to the individual who submitted the question. “I’ve never waited more than a day for a response,” said Senyak. The volunteer experts have even responded while on vacation, he said, adding that the answers are highly detailed and “powerful.”
All the questions, answers, and comments will eventually be published in the monthly MPN Clinic section of MPNforum Magazine. The entries will be archived in a searchable, publicly available online database.
The first MPN Clinic report will be published on September 15. However, Senyak said that responses to 27 questions have already been received and forwarded to patients in the United States and England. He is delighted by the volunteer experts’ helpfulness. “They have nothing to gain. It is purely altruistic of them,” he said.
MPNforum is financed by small donations, which, to date, amount to about $2000.
More About MPNs
MPNs are caused by one or more mutations, usually acquired after middle age. However, increasing numbers of younger adults and children are being diagnosed with the disorder, according to an MPNforum press statement. In MPNs, an overproduction of blood cells disrupts normal hematopoiesis. The various MPNs are differentiated by what is overproduced, such as erythrocytes in polycythemia vera or platelets in essential thrombocythemia.
If treated properly, many patients can live a normal life span with minimal suffering, according to the organization; however, some forms, such as myelofibrosis, can be debilitating and even fatal.
Myelofibrosis is associated with the dysregulation of 2 enzymes — janus-associated kinase (JAK)1 and 2 — which are involved in regulating blood and immunologic functioning. As the disease progresses, bone marrow is replaced with scar tissue, leading to anemia and thrombocytopenia. However, the scarred bone marrow tissue accumulates in other organs, most notably the spleen and liver. This collagen fibrosis can also cause bone pain, intense fatigue, and loss of appetite.
The only cure for myelofibrosis has been stem cell transplantation. However, since the implication of a genetic mutation in this disease was discovered, there have been developments in drug treatment. In 2011, the first drug treatment ever for myelofibrosis, ruxolitinib (Jakafi, Incyte Corp.), was approved by the US Food and Drug Administration.
Ruxolitinib has shown results that are “unprecedented” in the treatment of this disorder, according to Dr. Harrison, who is one of the volunteers and one of the drug’s principal investigators.
While research continues, access to MPN specialists remains the surest means of securing optimal treatment, said Senyak.