Therapy Can Prevent Infections in Immunocompromised Patients With Leukemia

Romyelocel-L, a treatment of ready-made human immune cells, can prevent infections in patients treated for leukemia who have a weakened immune system, according to the results of a phase 2 trial published in Journal of Clinical Oncology.

The researchers evaluated a single infusion of romyelocel-L in immunocompromised patients with acute myeloid leukemia (AML) undergoing intensive chemotherapy. Romyelocel-L consists of immature, human-derived immune cells that work to replenish neutrophils and help fight or prevent infections.

“The current standard of care (cytarabine- and anthracycline-based induction regimens) for younger and fit older patients results in serious infections and an early mortality rate of 7%-11% despite administration of antimicrobials,” the authors explained.

The researchers conducted a multicenter, randomized, open-label, phase 2 study of romyelocel-L plus granulocyte colony-stimulating factor (G-CSF; treatment arm) versus G-CSF monotherapy (control arm) in patients receiving induction chemotherapy. The goal was to reduce chemotherapy-induced neutropenia. Patients in the treatment arm received an infusion of romyelocel-L on day 9, 10, or 11.

A total of 163 patients were enrolled from 21 US centers, and 120 of the patients were evaluable. In the treatment arm, 59 patients were evaluable, and in the control arm, 61 patients were evaluable.

Only 6.8% of patients in the treatment group experienced infections compared with 27.9% in the control group. Patients in the treatment group spent less time in the hospital (average of 25.5 days) compared with the control group (average of 28.7 days).

“This cell therapy has great potential to help AML patients by reducing their infections, length of hospital stays, use of antibiotics, and development of antibiotic resistance—leading to improved outcomes for them overall,” said lead author Pinkal Desai, MD, assistant professor of medicine in the Division of Hematology and Medical Oncology; the Charles, Lillian, and Betty Neuwirth Clinical Scholar in Oncology at Weill Cornell Medicine; and an oncologist at NewYork-Presbyterian/Weill Cornell Medical Center.

Although the study met the end points showing lower incidence of infection, antibiotic use, and days of hospitalization in the treatment arm, there was no reduction in the mean duration of febrile episodes.

Patients receiving romyelocel-L had less frequent treatment-emergent significant adverse events (SAEs). Almost all of the SAEs (with the exception of 1) were considered not related to romyelocel-L. None of the patients on romyelocel-L had graft-versus-host disease or new-onset platelet refractoriness, nor did they have to be discontinued from the study because of an SAE or an AE.

“As infection remains the most common cause of morbidity and mortality in patients with AML, strategies to reduce the risk of infection are an unmet need,” the authors concluded. “These results provide the foundation for a phase 3 trial with the primary end point of reduction in infections during intensive induction chemotherapy for adults with AML.”

Reference

Desai PM, Brown J, Gill S, et al. Open-label phase II prospective, randomized, controlled study of romyelocel-L myeloid progenitor cells to reduce infection during induction chemotherapy for acute myeloid leukemia. J Clin Oncol. Published online June 22, 2021. doi:10.1200/JCO.20.01739