Robyn M. Emanuel1, Amylou C. Dueck1, Holly L. Geyer2,1, Jean‐Jacques Kiladjian1, Stefanie Slot1,3, Sonja Zweegman1,3, Peter te Boekhorst4,1, Suzan Commandeur5,1, Harry C. Schouten6,1, Federico Sackmann7,1, Ana Kerguelen Fuentes1, Dolores Hernández‐Maraver1, Heike L. Pahl1,8, Martin Grießhammer9,1, Frank Stegelmann1,10, Konstanze Doehner1,10, Thomas Lehmann1,11, Karin Bonatz1, Andreas Reiter1, Françoise Boyer1, Gabriel Étienne1, Jean‐Christophe Ianotto1, Dana Ranta1, Lydia Roy1, Jean‐Yves Cahn1, Claire Harrison12,1, Deepti Radia12,1, Pablo Muxí13,1, Norman Maldonado1,14, Carlos Besses1, Francisco Cervantes1, Peter Johansson15,1, Tiziano Barbui16,1, Giovanni Barosi17,1, Alessandro M. Vannucchi1,18, Francesco Passamonti1,19, Björn Andréasson15,1, Maria L Ferarri16,1, Alessandro Rambaldi16,1, Jan Samuelsson20,1, Gunnar Birgegård1,21, Ayalew Tefferi1, Ruben A. Mesa1
1Robyn M. Emanuel, Amylou C. Dueck, Holly L. Geyer, and Ruben A. Mesa, Mayo Clinic, Scottsdale, AZ; Jean-Jacques Kiladjian, Hopital Saint-Louis, Paris; Francoise Boyer, Centre Hospitalier Universitaire, Angers; Gabriel Etienne, Institut Bergonie, Bordeaux; Jean-Christophe Ianotto, Centre Hospitalier Universitaire, Brest; Dana Ranta, Hospitalier Universitaire, Nancy; Lydia Roy, Centre Hospitalier Universitaire, Poitiers; Jean-Yves Cahn, Centre Hospitalier Universitaire, Grenoble, France; Stefanie Slot and...
2Hospital Internal Medicine
3Vrije Universiteit, Amsterdam
4Erasmus University, Rotterdam
5Leiden University
6Maastricht Univ.
7Fundacion para Combatir La Leucemia
8Univ. of Freiburg
9Johannes Wesling Klinikum Minden
10Univ.of Ulm
11University of Basel
12Guy’s and St Thomas’ NHS Foundation Trust
13British Hospital Montevideo
14University of Puerto Rico
15NU Hospital Organization
16Ospedali Riuniti Di Bergamo
17IRCCS Fondazione Policlinico San Matteo
18University of Florence
19University of Pavia
20Karolinska Institutet
21 Uppsala University
Tóm tắt
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.
