Ethyol Pretreatment Reduces Gastrointestinal Side Effects of Bone Marrow Transplant in Myeloma Patients

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by Alice Melão |

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Ethyol HSCT toxicity

Pretreating multiple myeloma patients with Ethyol (amifostine) prior to a stem cell transplant significantly reduces the transplant therapy’s gastrointestinal toxicity without compromising its efficacy, a new study shows.

The study, “Amifostine reduces gastro-intestinal toxicity after autologous transplantation for multiple myeloma,” was published in the journal Leukemia & Lymphoma.

First described in 1983, the use of high-dose melphalan followed by autologous stem cell transplant remains one of the most effective therapies for the prevention of disease relapse in myeloma patients.

However, the treatment is associated with severe gastrointestinal side effects, including oral mucositis (inflammation and ulceration of the mucous membranes lining the mouth) anorexia, nausea, vomiting, and diarrhea.

These side effects are a major limitation to the use of stem cell transplants, especially in the elderly population, which constitute the majority of myeloma patients.

In an attempt to improve the outcomes of myeloma patients undergoing stem cell transplants, researchers at Case Western Reserve University in Cleveland, Ohio, studied whether Ethyol could prevent the gastrointestinal side effects of high-dose melphalan and stem cell transplant.

Ethyol, marketed by Cumberland Pharmaceuticals in the U.S., is approved by the U.S. Food and Drug Administration (FDA) for preventing cancer treatment toxicity. Small trials had suggested that Ethyol could reduce the gastrointestinal toxicity of melphalan.

The study included 107 myeloma patients who received two doses of Ethyol, given 24 hours and 15 minutes prior to high-dose melphalan and stem cell transplant, at University Hospitals Seidman Cancer Center.

As controls, the study used 114 patients from the MD Anderson Cancer Center who received high-dose melphalan and stem cell transplant without Ethyol pretreatment.

The frequency of all-grade oral mucositis, nausea, or vomiting was similar among the two groups. However, fewer Ethyol-treated patients had moderate to severe gastrointestinal symptoms.

While 86 percent of controls experienced moderate to severe nausea, only 32 percent of those who received Ethyol did. Similarly, oral mucositis, vomiting, and diarrhea were significantly less frequent in the Ethyol group.

Importantly, Ethyol did not influence the efficacy of stem cell transplant, with patients in both groups achieving similar overall survival and response rates.

Together, the findings may support the use of Ethyol for the prevention of gastrointestinal symptoms in myeloma patients undergoing stem cell transplants. Patients who are not currently considered suitable candidates due to their frail condition, including older patients, may soon have stem cell transplants included in their care.

“The confirmation of protective effect of [Ethyol] in prospective randomized trials along with a detailed cost analysis may lead to change in practice,” the researchers concluded.