Myeloproliferative neoplasm (MPN) symptom assessment form total symptom score: prospective international assessment of an abbreviated symptom burden scoring system among patients with MPNs.

Robyn M Emanuel , Amylou C Dueck , Holly L Geyer , Jean-Jacques Kiladjian (1) , Stefanie Slot , Sonja Zweegman , Peter a W Te Boekhorst , Suzan Commandeur , Harry C Schouten , Federico Sackmann , Ana Kerguelen Fuentes , Dolores Hernández-Maraver , Heike L Pahl , Martin Griesshammer , Frank Stegelmann , Konstanze Doehner , Thomas Lehmann (2) , Karin Bonatz , Andreas Reiter , Francoise Boyer , Gabriel Etienne , Jean-Christophe Ianotto (3) , Dana Ranta (4) , Lydia Roy (5) , Jean-Yves Cahn (6) , Claire N Harrison , Deepti Radia , Pablo Muxi , Norman Maldonado , Carlos Besses , Francisco Cervantes , Peter L Johansson , Tiziano Barbui (7) , Giovanni Barosi , Alessandro M Vannucchi , Francesco Passamonti , Bjorn Andreasson , Maria L Ferrari , Maria L Ferarri , Alessandro Rambaldi (8) , Jan Samuelsson , Gunnar Birgegard (9) , Ayalew Tefferi , Ruben A Mesa

1 Service d'hématologie clinique [Avicenne]
2 Paleontology section
3 Département d'Hématologie, Institut de Cancéro-hématologie
4 Service d'Hematologie et Médecine Interne
5 CIC 1402 - CIC Poitiers – Centre d'investigation clinique de Poitiers
6 TheREx
7 Hematology
8 Department of Oncology and Hematology
9 Department of Haematology, Division of Medicine, Institution for Medical Sciences

PURPOSE: Myeloproliferative neoplasm (MPN) symptoms are troublesome to patients, and alleviation of this burden represents a paramount treatment objective in the development of MPN-directed therapies. We aimed to assess the utility of an abbreviated symptom score for the most pertinent and representative MPN symptoms for subsequent serial use in assessing response to therapy. PATIENTS AND METHODS: The Myeloproliferative Neoplasm Symptom Assessment Form total symptom score (MPN-SAF TSS) was calculated as the mean score for 10 items from two previously validated scoring systems. Questions focus on fatigue, concentration, early satiety, inactivity, night sweats, itching, bone pain, abdominal discomfort, weight loss, and fevers. RESULTS: MPN-SAF TSS was calculable for 1,408 of 1,433 patients with MPNs who had a mean score of 21.2 (standard deviation [SD], 16.3). MPN-SAF TSS results significantly differed among MPN disease subtypes (P<.001), with a mean of 18.7 (SD, 15.3), 21.8 (SD, 16.3), and 25.3 (SD, 17.2) for patients with essential thrombocythemia, polycythemia vera, and myelofibrosis, respectively. The MPN-SAF TSS strongly correlated with overall quality of life (QOL; r=0.59; P<.001) and European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire C30 (EORTC QLQ-C30) functional scales (all P<.001 and absolute r≥0.50 except social functioning r=0.48). No significant trends were present when comparing therapy subgroups. The MPN-SAF TSS had excellent internal consistency (Cronbach's α=.83). Factor analysis identified a single underlying construct, indicating that the MPN-SAF TSS is an appropriate, unified scoring method. CONCLUSION: The MPN-SAF TSS is a concise, valid, and accurate assessment of MPN symptom burden with demonstrated clinical utility in the largest prospective MPN symptom study to date. This new prospective scoring method may be used to assess MPN symptom burden in both clinical practice and trial settings.

Domaines: Cancer

Robyn M Emanuel, Amylou C Dueck, Holly L Geyer, Jean-Jacques Kiladjian, Stefanie Slot, et al.. Myeloproliferative neoplasm (MPN) symptom assessment form total symptom score: prospective international assessment of an abbreviated symptom burden scoring system among patients with MPNs.. Journal of Clinical Oncology, 2012, 30 (33), pp.4098-103. ⟨10.1200/JCO.2012.42.3863⟩. ⟨hal-00849679⟩