Symptomatic Profiles of Patients With Polycythemia Vera: Implications of Inadequately Controlled Disease.

Holly Geyer , Robyn Scherber , Heidi Kosiorek , Amylou C Dueck , Jean-Jacques Kiladjian (1, 2) , Zhijian Xiao , Stefanie Slot , Sonja Zweegman , Federico Sackmann , Ana Kerguelen Fuentes , Dolores Hernández-Maraver , Konstanze Döhner , Claire N Harrison , Deepti Radia , Pablo Muxi , Carlos Besses , Francisco Cervantes , Peter L Johansson , Bjorn Andreasson , Alessandro Rambaldi (3) , Tiziano Barbui (4) , Karin Bonatz , Andreas Reiter , Francoise Boyer (5, 6) , Gabriel Etienne (7) , Jean-Christophe Ianotto (8, 9) , Dana Ranta , Lydia Roy , Jean-Yves Cahn (10) , Norman Maldonado , Giovanni Barosi , Maria L Ferrari (11) , Robert Peter Gale , Gunnar Birgegard (12) , Zefeng Xu (13) , Yue Zhang , Xiujuan Sun , Junqing Xu (14) , Peihong Zhang (15) , Peter a W Te Boekhorst , Suzan Commandeur , Harry Schouten , Heike L Pahl , Martin Griesshammer , Frank Stegelmann , Thomas Lehmann , Zhenya Senyak , Alessandro M Vannucchi , Francesco Passamonti , Jan Samuelsson , Ruben A Mesa

1 Hématologie -Immunologie -Cibles thérapeutiques
2 Centre d'Investigations Cliniques
3 Hematology and Bone Marrow Transplant Unit
4 Hematology
5 SPVQVRSF - Santé publique, vieillissement, qualité de vie et réadaptation des sujets fragiles - EA 3797
6 Service de Médecine Physique Neurologique-Traumatologique [Reims]
7 IPBS - Institut de pharmacologie et de biologie structurale
8 Institut de cancérologie et d'hématologie
9 GETBO - Groupe d'Etude de la Thrombose de Bretagne Occidentale
10 TheREx
11 LRGP - Laboratoire Réactions et Génie des Procédés
12 Department of Haematology, Division of Medicine, Institution for Medical Sciences
13 LBMCE - Laboratoire de Biologie Moléculaire et Cellulaire des Eucaryotes
14 IMPLAD - Institute of Medicinal Plant Development
15 State Key Laboratory of Rare Resource Utilization

Polycythemia vera (PV) is a myeloproliferative neoplasm (MPN) associated with disabling symptoms and a heightened risk of life-threatening complications. Recent studies have demonstrated the effectiveness of JAK inhibitor therapy in patients with PV patients who have a history of prior hydroxyurea (HU) use (including resistance or intolerance), phlebotomy requirements, and palpable splenomegaly. We aimed to determine how these features contribute alone and in aggregate to the PV symptom burden. Through prospective evaluation of 1,334 patients with PV who had characterized symptom burden, we assessed patient demographics, laboratory data, and the presence of splenomegaly by disease feature (ie, known HU use, known phlebotomy requirements, splenomegaly). The presence of each feature in itself is associated with a moderately high symptom burden (MPN symptom assessment form [SAF] total symptom score [TSS] range, 27.7 to 29.2) that persists independent of PV risk category. In addition, symptoms incrementally increase in severity with the addition of other features. Patients with PV who had all three features (PV-HUPS) faced the highest total score (MPN-SAF TSS, 32.5) but had similar individual symptom scores to patients with known HU use (PV-HU), known phlebotomy (PV-P), and splenomegaly (PV-S). The results of this study suggest that patients with PV who have any one of the features in question (known HU use, known phlebotomy, or splenomegaly) have significant PV-associated symptoms. Furthermore, it demonstrates that many PV symptoms remain severe independent of the number of features present.

Mots clés: Polycythemia Vera; Symptomatic Profiles

Domaines: Médecine humaine et pathologie; Sciences du Vivant [q-bio]

Holly Geyer, Robyn Scherber, Heidi Kosiorek, Amylou C Dueck, Jean-Jacques Kiladjian, et al.. Symptomatic Profiles of Patients With Polycythemia Vera: Implications of Inadequately Controlled Disease.. Journal of Clinical Oncology, 2016, 34 (2), pp.151-9. ⟨10.1200/JCO.2015.62.9337⟩. ⟨hal-01276589⟩