Patient-Reported Outcomes (PROs) are patient-centered measures used to assess health status, track changes over time, and evaluate the impact of treatment on the patient's perceived health.
The SACHA study is a French prospective observational study developed by the Société Française de lutte contre les Cancers de l'Enfant et de l'adolescent (SFCE). It prospectively collects real-world safety and activity data on novel therapies given to patients aged 25 or younger with pediatric malignancies (solid tumors or hematologic malignancies) or related conditions, outside of a clinical trial.
The Symptom Screening in Pediatrics Tool (SSPedi) is a validated questionnaire for measuring patient-reported symptoms in pediatric oncology. It includes 15 questions covering common symptoms in pediatric cancer patients and one open-ended question allowing patients to report any other bothersome symptoms. Patients complete the questionnaire through an online application.
The PRO-SACHA study aims to describe the symptoms reported by participants receiving novel therapies in pediatric oncology and to examine the concordance between participant-reported symptoms and symptomatic adverse events (AEs) reported by investigators.
This prospective observational study evaluates patient-reported symptoms in patients aged 2 to 18 years enrolled in the SACHA study. Participation is voluntary, based on an opt-out consent model (French category 3 interventional research involving the human person).
Planned enrollment: 72 participants over 18 months, with each participant followed for 7 months (a 6-month follow-up period, with a 7th-month window for questionnaire completion). Participants are enrolled in PRO-SACHA at the same time as their enrollment in SACHA (two separate studies with separate enrollment).
SSPedi responses (a self-report questionnaire capturing symptoms experienced by patients) are collected electronically through an online application. The extracted, anonymized data are then correlated with adverse events reported by investigators. The concordance between participant-reported symptoms and adverse events recorded in SACHA (CTCAE grading) will be analyzed.
Aged 2 to 18 years
Patients aged 8 to 18 years who can read French, English, or Spanish fluently OR patients aged 6 to 7 years who can read French, English, or Spanish fluently, OR patients aged 2 to 7 years who cannot read fluently but whose parents can read French, English, or Spanish fluently
Patients included in the SACHA study before starting treatment
Patients with a pediatric tumor or leukemia who have failed treatment or relapsed and have no standard treatment options available OR patients receiving first-line treatment with no standard treatment options available (e.g., inoperable plexiform neurofibroma and MEK inhibitors, infantile fibrosarcoma and NTRK inhibitors)
Patient who has already started the innovative treatment
Patients aged 8 years and older who cannot read
Patients whose clinical condition does not allow them to report symptoms themselves (i.e., non-communicative patient; judgment left to the investigator's discretion)
Patients currently enrolled in an early-phase trial
Assistance Publique Hopitaux De Marseille
Lead sponsor
The Hospital for Sick Children
Collaborator
Gustave Roussy, Cancer Campus, Grand Paris
Collaborator