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


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.


CONCLUSIONS
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.
Division of Hematology and Oncology, Mayo Clinic, 13400 E. Shea Blvd, Scottsdale, AZ 85259, USA.
Robyn M
RM
Emanuel
Amylou C
AC
Dueck
Holly L
HL
Geyer
Jean-Jacques
JJ
Kiladjian
Stefanie
S
Slot
Sonja
S
Zweegman
Peter A W
PA
te Boekhorst
Suzan
S
Commandeur
Harry C
HC
Schouten
Federico
F
Sackmann
Ana
A
Kerguelen Fuentes
Dolores
D
Hernández-Maraver
Heike L
HL
Pahl
Martin
M
Griesshammer
Frank
F
Stegelmann
Konstanze
K
Doehner
Thomas
T
Lehmann
Karin
K
Bonatz
Andreas
A
Reiter
Francoise
F
Boyer
Gabriel
G
Etienne
Jean-Christophe
JC
Ianotto
Dana
D
Ranta
Lydia
L
Roy
Jean-Yves
JY
Cahn
Claire N
CN
Harrison
Deepti
D
Radia
Pablo
P
Muxi
Norman
N
Maldonado
Carlos
C
Besses
Francisco
F
Cervantes
Peter L
PL
Johansson
Tiziano
T
Barbui
Giovanni
G
Barosi
Alessandro M
AM
Vannucchi
Francesco
F
Passamonti
Bjorn
B
Andreasson
Maria L
ML
Ferrari
Maria L
ML
Ferarri
Alessandro
A
Rambaldi
Jan
J
Samuelsson
Gunnar
G
Birgegard
Ayalew
A
Tefferi
Ruben A
RA
Mesa
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009196:Myeloproliferative Disorders / Q000175:diagnosis* / Q000473:pathology; D011446:Prospective Studies; D011788:Quality of Life; D012720:Severity of Illness Index; D018434:Sickness Impact Profile; D011795:Surveys and Questionnaires; D055815:Young Adult