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Endocrine Abstracts (2022) 84 PS3-15-134 | DOI: 10.1530/endoabs.84.PS3-15-134

ETA2022 Poster Presentations Thyroid Cancer Diagnosis & Treatment (9 abstracts)

Patient-reported outcomes (PROS) from patients with RET-mutant medullary thyroid cancer (MTC) and ret fusion-positive TC treated with pralsetinib in the arrow trial

Julien Hadoux 1 , Mimi I Hu 2 , Marcia S. Brose 3 , Elena Garralda 4 , Christine Spitzweg 5 , Noemí Reguart 6 , Christine Do Cao 7 , Martin D. Forster 8 , Teresa Alonso-Gordoa 9 , Teresa Barata 10 , Astrid Scalori 11 , Alena Zalutskaya 12 , Peter Trask 13 & Vivek Subbiah 2


1Gustave Roussy, Villejuif, France; 2The University of Texas MD Anderson Cancer Center, Houston, United States; 3Sidney Kimmel Cancer Center, Jefferson University, Philadelphia, Pa, United States; 4Vall D’ Hebron Institute of Oncology, Barcelona, Spain; 5University Hospital, LMU Munich, Department of Medicine IV, Munich, Germany; 6Hospital Clínic, Barcelona, Spain; 7Hôpital Claude Huriez, Lille, France; 8Ucl Cancer Institute/University College London Hospitals, London, United Kingdom; 9Hospital Universitario Ramón Y Cajal, Universidad de Alcalá, Madrid, Spain; 10F. Hoffmann-La Roche Ltd, Basel, Switzerland; 11F. Hoffmann-La Roche Ltd, Welwyn Garden City, United Kingdom; 12Blueprint Medicines Corporation, Cambridge, Massachusetts, United States; 13Genentech, Inc., South San Francisco, Ca, United States


Objectives: RET alterations are targetable oncogenic drivers in TC. Patients with TC, especially those with MTC treated with the multikinase inhibitors cabozantinib and/or vandetanib (C/V), often experience significant treatment-related side effects. Pralsetinib, a selective RET tyrosine kinase inhibitor, showed efficacy in patients with RET-altered TC from the phase 1/2 ARROW trial (NCT03037385). We present the impact of pralsetinib on PROs in patients with RET-mutant MTC and RET fusion-positive TC, including quality of life (QoL) and disease-related symptoms.

Methods: PROs (exploratory endpoint since protocol v4.1) were evaluated in adults with RET-altered, non-resectable advanced TC from ARROW (pralsetinib 400 mg QD) who completed the European Organization for Research and Treatment of Cancer (EORTC) QoL questionnaire (QLQ-C30) before treatment initiation (baseline). Patients then completed the QLQ-C30 every 4 weeks until treatment discontinuation. Score changes >10 points from baseline were considered clinically meaningful. Data cut-off: 18 October 2021.

Results: Of 100 efficacy-evaluable patients with RET-mutant MTC enrolled from protocol v4.1, 98 (98%) completed a baseline PRO assessment. They reported moderate baseline Global Health Status (GHS)/QoL (mean score: 67/100) and high baseline functioning scores (mean scores: >79/100), with no clinically meaningful changes from baseline to Week 44 (≥25% of patients still enrolled). Mean score for diarrhoea improved from baseline to Week 44 (–25.9). In the prior C/V RET-mutant MTC subcohort (n = 39), baseline mean GHS/QoL score was 59/100 and functioning scores were >68/100. Clinically meaningful increases in mean scores from baseline were observed throughout Weeks 8–32 for GHS/QoL, Weeks 24–32 for physical functioning and Weeks 12–44 for role functioning. At Week 44, disease-related symptoms including diarrhoea (mean score change from baseline: –20.6), fatigue (–14.8), appetite loss (–12.7) and insomnia (–12.7) were improved. Patients with treatment-naïve RET-mutant MTC (n = 54) had higher baseline GHS/QoL (mean score: 71/100) and functioning scores (mean scores: >82/100) than those who had received prior C/V, and their PROs remained stable following pralsetinib treatment. In patients with RET fusion-positive TC enrolled from protocol v4.1 (n = 24), baseline PRO questionnaire completion rate was 100%. At baseline, these patients had moderate GHS/QoL (mean score: 59/100), and moderate-to-high functioning scores (mean scores: >70/100); episodic clinically meaningful improvements were seen for GHS/QoL and role functioning, and symptom burden remained low throughout.

Conclusions: Following pralsetinib treatment, patients with RET-altered TC, especially those with RET-mutant MTC treated with prior C/V, reported improved or stable GHS/QoL and functioning scores, and a reduced symptom burden from baseline.

Volume 84

44th Annual Meeting of the European Thyroid Association (ETA) 2022

Brussels, Belgium
10 Sep 2022 - 13 Sep 2022

European Thyroid Association 

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