Substudy to Assess CML Patients’ Perspectives on Treatment-Free Remission

A host of emotions may affect a person who is choosing to discontinue ongoing therapy for chronic myeloid leukemia (CML) upon achieving deep molecular response (DMR). But there’s not a substantial body of research that’s collected and analyzed those reactions.

A team of researchers hopes to expand the existing body of research by embarking upon a new project to learn more about the perspectives and emotional experiences of patients with Philadelphia chromosome-positive (Ph+) CML both during and after the cessation of therapy with nilotinib, a second-generation tyrosine kinase inhibitor (TKI). They detailed an early present of their research, which is a psychological substudy of a larger phase 3 study known as ENESTPath, in a recent article in the journal Frontiers in Oncology.

This Italian substudy will analyze the perspectives of patients with Ph+ CML participating in the ENESTPath study. Participants in ENESTPath received nilotinib 300 mg twice daily for a 12-month induction phase followed by a 12-month consolidation phase with nilotinib. Participants who achieved DMR were randomly assigned to immediate discontinuation of treatment (a total of 24 months of nilotinib therapy) or an additional consolidation period of 12 months, for a total of 36 months of nilotinib therapy.

The substudy is a mixed method study using both quantitative and qualitative methods to assess participants’ emotions and experiences. Quantitative methods will be used for psychological assessment of patients in the consolidation group, who also will be asked to keep a diary for qualitative insights. Unstructured interviews will be used to assess those in the TFR group. Both groups of patients will be assessed at various times for health-related quality of life (HRQoL) outcomes.

The researchers noted that clinical practice guidelines for CML treatment do not currently incorporate psychological or emotional issues related to stopping TKI treatment and attempting TFR. But these approaches “hold great potential for the improved management of CML in the near future,” driving the need for more insight into the patient perspective.

“The CML patients’ voice substudy is unique as it is designed for in-depth assessment of a wide range of possible psychological and emotional variables along with an inner understanding of the patient’s experience directly from [their] perspective and words, that might provide better clinical practice insights for informing future discussions with patients who are considering treatment termination,” the researchers wrote, adding that the insights might also help in the design of future studies involving CML treatment discontinuation.

“These insights could help clinicians in tailoring their communication and interaction with patients, having in mind the complexity of the patients’ emotional experience,” they concluded.

Disclosure: This research was supported by Novartis Pharma AG. Some study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures.

Reference

Borghi L, Rosti G, Maggi A, et al. Perspectives and emotional experiences of patients with chronic myeloid leukemia during ENESTPath clinical trial and treatment-free remission: rationale and protocol of the Italian substudy. Front Oncol. 2021;11:638689. doi:10.3389/fonc.2021.638689