POEMS: An expertise in the rare leads to a rare opportunity

In the medical community, POEMS does not stand for the famous works of Robert Frost but a rare blood disorder syndrome that damages your nerves and affects many other parts of the body. POEMS stands for these signs and symptoms:

• Polyneuropathy
  Numbness, tingling and weakness in your legs — and, over time, maybe in your hands — and difficulty breathing.
• Organomegaly
  Enlarged spleen, liver or lymph nodes.
• Endocrinopathy
  Abnormal hormone levels that can result in underactive thyroid (hypothyroidism), diabetes, sexual problems, fatigue, swelling in your limbs, and problems with metabolism and other essential functions.
• Monoclonal plasma-proliferative disorder
  Abnormal bone marrow cells (plasma cells) that produce a protein (monoclonal protein) that can be found in the bloodstream.
• Skin changes
  More color than normal on your skin, possibly thicker skin and increased facial or leg hair.

Because of these varying symptoms, Angela Dispenzieri, M.D., a Mayo Clinic hematologist and POEMS expert, says that POEMS often is misdiagnosed.

“There are so many symptoms that would seem to be unrelated, who would ever think they are all connected,” Dr. Dispenzieri says. “The fact is all of these symptoms are driven by a bone marrow process.”

Dr. Dispenzieri’s expertise started in the late 1990s, when she wrote a paper about the rare syndrome.

“I didn’t know much about POEMS until I started writing about it,” Dr. Dispenzieri says. “I did my research, and, before I knew it, patients started coming to me. The more patients I saw, the more expert I became, and my reputation grew.”

Dr. Dispenzieri’s reputation has now grown to the point of international recognition for diagnosis and treatment of POEMS. As a result, Dr. Dispenzieri received an unexpected gift and opportunity from an unexpected source.

“A patient with POEMS went to a treatment center abroad that suggested the patient confer with me,” Dr. Dispenzieri says. “This patient contacted me and said that they would like to fund research for the future of treatment, despite never being a patient at Mayo Clinic ─ simply because they were told it was the best place to be treated.”

The patient donated several million to fund research and a clinical trial for the treatment of POEMS. The contract includes subsidies for the cost of travel, lodging and support for the participants.

According to Dr. Dispenzieri, this clinical trial is important because it allows the use of well-researched drug treatments for multiple myeloma in the use of treatment for POEMS “One combination that looks good in multiple myeloma is a three-drug combination of ixazomib, lenalidomide, and dexamethasone, and we wanted to try that combo in patients with POEMS.”

The clinical trial, currently in open enrollment, will include 34 patients, with half newly diagnosed and half who have had prior therapy and relapsed. Patients will undergo multiple tests, most of which are paid for by the generous gift. Newly diagnosed patients receive the treatment for three months and are then reassessed before they move on to an autologous peripheral blood stem cell transplant. Relapsed patients receive the drug for one year.

Dr. Dispenzieri says she is honored to be recognized with clinical trial funding.

“We are the land of the rare ─ a place of last resort for many patients,” Dr. Dispenzieri says. “To receive a donation from an individual who was not a patient of Mayo Clinic and based solely on the recognition of our specialty is humbling.”

Learn more about clinical trials at Mayo Clinic and the inclusion criteria.