Back
The Myleloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) Derived Total Symptom Score (TSS): An International Trial of 1433 Patients with Myeloproliferative Neoplasms (MPNs),
Journal article

The Myleloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) Derived Total Symptom Score (TSS): An International Trial of 1433 Patients with Myeloproliferative Neoplasms (MPNs),

  • Scherber, Robyn Chicago Medical School, Rosalind Franklin School of Medicine, Chicago, IL, USA,
  • Dueck, Amylou Mayo Clinic, Scottsdale, AZ, USA,
  • Kiladjian, Jean-Jacques Centre d'Investigations Cliniques, Hopital Saint-Louis; and FIM (French Intergroup of Myeloproliferative disorders), Paris, France,
  • Slot, Stephanie Department of Hematology, VU University Medical Center, Amsterdam, Netherlands,
  • Zweegman, Sonja Dept. of Hematology, VUMC, Amsterdam, Netherlands,
  • te Boekhorst, Peter Erasmus Medical Center, Rotterdam, Netherlands,
  • Commandeur, Suzan Department of Dermatology, LUMC, Leiden, Netherlands,
  • Schouten, Harry C. Academische Ziekenhuis Maastricht, Maastricht, Netherlands,
  • Sackmann, Federico GATLA, Grupo Argentino de Tratamiento de Leucemia Aguda, Buenos Aires, Argentina,
  • Fuentes, Ana Kerguelen Department of Haematology, University Hospital La Paz, Madrid, Spain,
  • Hernandez-Maraver, Dolores Hematology, La Paz University Hospital, Madrid, Spain,
  • Pahl, Heike L. Clinical Research Center Experimental Anaesthesiology, University Hospital Freiburg, Freiburg, Germany,
  • Griesshammer, Martin Hematology and Oncology, Johannes Wesling Clinic Minden, Minden, Germany,
  • Stegelmann, Frank Department of Internal Medicine III, University Hospital of Ulm, Ulm, Germany,
  • Doehner, Konstanze Dept. Internal Medicine III, University of Ulm, Ulm, Germany,
  • Lehmann, Thomas Hematology, University Hospital Basel, Basel, Switzerland,
  • Bonatz, Karin Medizinische Klinik, Universitätsmedizin Mannheim, Mannheim, Germany,
  • Reiter, Andreas III. Medizinische Klinik, Universitätsmedizin Mannheim, Mannheim, Germany,
  • Boyer, Françoise Service d'Hématologie Clinique, Centre Hospitalier Universitaire, Angers, France,
  • Etienne, Gabriel Institut Bergonié, Bordeaux,
  • Ianotto, Jean-Christophe Clinical Hematology, Brest University Hospital, Brest, France,
  • Ranta, Dana Department of Hematology, Nancy University Hospital, Vandoeuvre-Les-Nancy, France,
  • Roy, Lydia Blood Diseases, Poitiers University Hospital, Poitiers, France,
  • Cahn, Jean-Yves Onco-Hematologie, CHU-Grenoble, Grenoble, Cedex O9, France,
  • Harrison, Claire N. Dept. of Haematology, Guy's and St. Thomas NHS FoundationTrust, London, United Kingdom,
  • Radia, Deepti Dept. of Haematology, Guys and St Thomas NHS Foundation Trust, London, United Kingdom,
  • Muxi, Pablo J. Unidadde Hematología, Hospital Británico, Montevideo, Uruguay,
  • Maldonado, Norman I School of Medicine, University of Puerto Rico, San Juan, PR, PR,
  • Besses, Carlos Hematology, Hospital del Mar, Barcelona, Spain,
  • Cervantes, Francisco Dept. of Hematology, Hospital Clinic, Barcelona, Spain,
  • Johansson, Peter Department of Medicine, Hematology and Coagulation Section, Gothenburg, Sweden,
  • Barbui, Tiziano USC Hematology, Ospedali Riuniti di Bergamo, Bergamo, Italy,
  • Barosi, Giovanni Clinical Epidemiology/Center for the Study of Myelofibrosis, IRCCS Policlinico S.Matteo Foundation, Pavia, Italy,
  • Vannucchi, Alessandro M. Hematology, University of Florence, Florence, Italy,
  • Passamonti, Francesco Department of Hematology, Fondazione IRCCS Policlinico San Matteo, University of Pavia, Pavia, Italy,
  • Andreasson, Bjorn Internal Medicine, NU Hospital Organization, Uddevalla, Sweden,
  • Ferarri, Maria L Biol. Sci., Ospedali Riuniti di Bergamo, Bergamo, Italy,
  • Rambaldi, Alessandro USC Hematology, Ospedali Riuniti di Bergamo, Bergamo, Italy,
  • Samuelsson, Jan Department of Internal Medicine, Stockholm South Hospital, Stockholm, Sweden,
  • Birgegard, Gunnar Dept. of Hematology, University Hospital, Uppsala, Sweden,
  • Tefferi, Ayalew Division of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, MN, USA,
  • Mesa, Ruben A. Division of Hematology and Medical Oncology, Mayo Clinic, Scottsdale, AZ, USA
Show more…
Published in:
  • Blood. - American Society of Hematology. - 2011, vol. 118, no. 21, p. 3839-3839
Immunology
Cell Biology
Biochemistry
Hematology
English Abstract
Abstract 3839

BACKGROUND:
We have previously reported on the validation of the 18 item Myeloproliferative Neoplasm Assessment Form (MPN-SAF) (Blood 2011;118:401–408) given in conjunction with the 9 item Brief Fatigue Inventory (BFI) (Cancer 1999;85:1186–1196) to assess symptomatic burden in an international sample of MPN patients (pts), including validation in English, Italian, Swedish, German, French, Spanish, and Dutch. We desired to assess the utility of an average total symptom score (TSS) from the most pertinent and representative MPN symptoms for purposes of assessing the burden of symptoms in MPN pts, and subsequent tracking in response to therapy.


METHODS:
Data was collected among an international cohort of MPN pts and their physicians, including patient demographics and disease features and completion the BFI, MPN-SAF and the EORTC-QLQ-C30. Among pts who completed at least 5 of 10 specific items on the BFI and MPN-SAF, an average score was calculated as the TSS. TSS items included “worst” fatigue from the BFI and 9 items from the MPN-SAF including concentration, early satiety, inactivity, night sweats, itching, bone pain, abdominal discomfort, weight loss and fever. The TSS thus had a possible range of 0–10 with 10 representing the highest level of symptom severity. Data was then analyzed for internal consistency, and divergent, convergent validity, and construct validity.


RESULTS:
Patient Demographic and Disease Characteristics:1433 MPN pts were prospectively enrolled (Argentina 22, France 482, Germany 59, Italy 186, Netherlands 236, Puerto Rico 10, United Kingdom 57, United States 102, Spain 157, Sweden 114, Uruguay 8) including 594 ET, 538 PV and 293 MF pts (8 missing; MF: 61% Primary MF, 23% post-ET MF, 15% post-PV MF). 1408 pts completed at least 5 of the 10 items necessary to calculate a TSS. Pts were of characteristic age (mean 62, range 20–94) and gender (54% female) common to disease. TSS Burden of MPN Symptoms: Consistent with prior studies, the majority of pts (>50%) were symptomatic in each TSS item except for items associated with high disease severity, namely bone pain (48.6%), weight loss (30.6%) and fever (18.4%). Fatigue carried the highest symptom intensity (4.4, SD=2.8), followed by problems with concentration (2.5, SD=2.8) and early satiety (2.5, SD=2.7). Overall mean TSS was 2.1 (SD=1.6). Divergent Validity: TSS significantly differed among MPN disease subtypes (p<0.001) with means of 1.9 (SD=1.5), 2.2 (SD=1.6), and 2.5 (SD=1.7) for ET, PV, and MF pts, respectively. Statistically significant differences in TSS were also observed between pts with clinically deficient (>4, n=480) versus non-clinically deficient QOL (<4, n=894; mean 3.3 versus 1.5; p<0.001). When comparing to MD perceptions, TSS was significantly higher when MDs rated >2 of 6 common MPN-related symptoms as clinically significant (2.8, n=400) versus <2 symptoms (1.6, n=726; p<0.001). No significant trends were observed when comparing disease type by the presence of a current medical therapy. Convergent Validity: The TSS was strongly correlated with patient-reported QOL (r=0.59, p<0.001). Overall excellent correlations existed between the TSS and EORTC-QLQ-C30 functional subscales (all p<0.001 and r>0.50 except social functioning [r=0.48]). Additionally, excellent correlations were observed between the TSS and EORTC-QLQ-C30 fatigue and pain symptom scales (r>0.5, p<0.001). Internal Consistency and Construct Validity: The TSS had excellent internal consistency (Cronbach's alpha=0.83). Factor analysis identified a single underlying construct among the 10 TSS items (significant eigenvalues being >1). Factor loadings ranged from 0.43 for fever and weight loss to 0.71 for inactivity. The single factor suggests that the arithmetic mean of the 10 items is an appropriate global TSS score.


CONCLUSION:
The TSS demonstrated excellent psychometric properties. Overall, results of validity and internal consistency indicate that the TSS is a concise, valid, and accurate assessment of symptom burden among MPN pts. This new scoring will facilitate ease of implementation of the MPN-SAF into larger clinical trials and reduce ambiguity associated with interpreting response outcomes. Future analyses to investigate the impact of therapies on TSS are ongoing.


Disclosures:
No relevant conflicts of interest to declare.
Language
  • English
Open access status
closed
Identifiers
Persistent URL
https://folia.unifr.ch/global/documents/94240
Statistics
Document views: 82 File downloads: