A real-world evidence study in Locally Advanced Head and Neck Squamous Cell Carcinoma (LA HNSCC), across Europe

Objectif(s) de la recherche et intérêt pour la santé publique

Finalité de l'étude

Objectifs poursuivis

Domaines médicaux investigués

Bénéfices attendus

Public Heath Interest:

More than half of patients present with locally advanced (LA) disease. If their tumors are resectable, surgery with curative intent (followed by appropriate adjuvant therapy) is preferred. However, in unresectable LA HNSCC, where the tumors are not amendable to curative surgery or where prognosis is anticipated to be poor or result in unacceptable functional, outcomes platinum based chemoradiotherapy (CRT) is the standard of care (SoC). In a retrospective study on 84,046 patients with HNSCC, the use of radiotherapy (RT) increased over time, with platinum based CRT remaining the most commonly used regimen, though less frequently in patients aged >70 years. Platinum based CRT was associated with more favorable five year overall survival (OS) than alternative RT based regimens across age groups, but is accompanied by substantial acute and long term toxicities. A significant proportion of patients are ineligible for platinum therapy (e.g. high-dose cisplatin) due to comorbidities or organ dysfunction. For these cases, guidelines recommend alternative regimens such as RT plus cetuximab or carboplatin with 5-fluorouracil (5 FU) but RT alone often remains as the default option despite inferior efficacy compared to CRT. To address this unmet need, JNJ-1900, a novel intratumoral radioenhancer, is undergoing clinical development. To test the new treatment’s efficacy, Johnson & Johnson is conducting NANORAY 312, a phase III trial evaluating JNJ-1900 plus RT versus RT alone, with optional cetuximab, in patients ≥60 years with LA HNSCC who are ineligible for platinum based CRT.

Research Question:

The purpose of this study is to describe RW baseline characteristics, treatment patterns, clinical outcomes, health care resource utilization (HCRU), and patient reported outcomes (PROs) among patients in Europe with treated LA HNSCC who did not undergo surgery as initial treatment (No Surgery Cohort). These outcomes will be evaluated overall and within the following pre specified cohorts: patients who were exposed to platinum based chemotherapy (Platinum Exposed Cohort), patients who were not exposed to platinum based therapy (Platinum Unexposed Cohort), and patients who were not exposed to platinum-based therapy who were also eligible for the NANORAY 312 study (NANORAY Eligible Cohort).

Objectives:

Primary Objective:
- Describe patient outcomes over follow-up.

Secondary Objectives:
- Describe baseline demographic and clinical characteristics of patients at index
- Characterize RW SoC treatment patterns

Exploratory objectives:
- Describe baseline demographic and clinical characteristics at index among patients who have either HCRU or PROs recorded
- Describe HCRU and PRO outcomes over follow-up

Method:

This is a retrospective, non-interventional cohort study that utilizes secondary data from hospital sites’ electronic medical records (EMRs) to generate RW evidence in support of contextualizing the findings of J&J’s NANORAY-312 trial. The study cohorts will comprise of patients with LA HNSCC who meet the eligibility criteria and are identified across all study sites included in the site selection framework.

Study Population:

The patients will be classified into the four relevant study cohorts, as listed below:

• No-Surgery and Treated Cohort: Patients with LA HNSCC who did not receive surgery with curative intent as their first anti-cancer treatment.
• Platinum-Exposed Cohort: Patients in the No-Surgery and Treated Cohort who initiated platinum-based chemotherapy as (part of) their first anti-cancer treatment.
• Platinum-Unexposed Cohort: Patients who did not receive surgery with curative intent as their first anti-cancer treatment and did not initiate platinum-based chemotherapy as (part of) their first anti-cancer treatment.
• NANORAY-Eligible Cohort: Patients in the Platinum‑Unexposed Cohort who met selected eligibility criteria for the NANORAY trial, as determined using RW proxy variables designed to approximate the trial eligibility criteria.

For all main analyses of this study, the Platinum‑Unexposed Cohort will comprise of patients with no exposure to any platinum‑based therapy (i.e., neither cisplatin nor carboplatin, irrespective of dose) as part of their first anti‑cancer treatment. However, a sensitivity analysis will be conducted using an alternative operational definition for the Platinum‑Unexposed Cohort.