05:09am Thursday 21 September 2017

Powerful Three-drug Combo Shows Promise for People with Rare Blood Disease

AL amyloidosis is an incurable disease in which abnormal proteins produced in the bone marrow travel to major organs of the body, leaving deposits that typically damage the heart and kidneys. The median overall survival for those with this disease is approximately three years. For those where the disease causes heart damage, the overall survival is less than a year.

“This novel drug combination was able to effectively shut off the production of the abnormal proteins,” says the study’s lead researcher Joseph Mikhael, M.D., a hematologist at Mayo Clinic in Arizona.

Click here as Dr. Mikhael explains the study.

Prior to the CyBorD regimen, those with AL amyloidosis had few effective treatment options to keep the disease in check, explains Dr. Mikhael. “What we found remarkable was how fast the combination worked — the average time to response was less than two months,” he says. “Furthermore, over 90 percent of patients had at least a 50 percent drop in the protein, with over 70 percent having the protein disappear entirely.”

Typically, treatment includes a lengthy regimen of chemotherapy and possibly a stem cell transplant for those few patients who meet the medical criteria to receive a transplant.

CyBorD treatment consists of the drugs cyclophosphamide, bortezomib and dexamethasone. The research found that this treatment:

  • Rapidly reduced the amount of abnormal proteins
  • Was easier for patients to tolerate, compared to the previous chemotherapy protocol used
  • Could be used in patients who were eligible and ineligible for transplant
  • Was effective as a first-line treatment and also in relapsed disease

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