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Myeloproliferative (MPN) Symptom Burden Response Thresholds: Assessment Of MPN-SAF TSS Quartiles As Potential Markers Of Symptom Response
Journal article

Myeloproliferative (MPN) Symptom Burden Response Thresholds: Assessment Of MPN-SAF TSS Quartiles As Potential Markers Of Symptom Response

  • Emanuel, Robyn M. Internal Medicine, Mayo Clinic, Scottsdale, AZ, USA,
  • Dueck, Amylou Constance Mayo Clinic Arizona, Scottsdale, AZ, USA,
  • Geyer, Holly Lynn Internal Medicine, Mayo Clinic, Scottsdale, AZ, USA,
  • Kiladjian, Jean-Jacques Hôpital Saint-Louis et Université Paris Diderot, Paris, France,
  • Slot, Stephanie Department of Hematology, VU University Medical Center, Amsterdam, Netherlands,
  • Zweegman, Sonja Department of Hematology, VU University Medical Center, Amsterdam, Netherlands,
  • Boekhorst, Peter te 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,
  • Hernandez, Dolores Hematology, Hospital Universitario La Paz, Madrid, Spain,
  • Fuentes, Ana Kerguelen Department of Haematology,, University Hospital La Paz, Madrid, Spain,
  • Pahl, Heike L. Department of Hematology/Oncology, University Hospital Freiburg, Freiburg, Germany,
  • Stegelmann, Frank Department of Internal Medicine III, University Hospital of Ulm, Ulm, Germany,
  • Doehner, Konstanze Department of 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 Hämatologie und Onkologie, Universitätsmedizin Mannheim, III. Medizinische Klinik, Mannheim, Germany,
  • Boyer, Françoise Centre Hospitalier Universitaire, Angers, France,
  • Etienne, Gabriel Institut Bergonié, Bordeaux, Bordeaux, France,
  • Ianotto, Jean-Christophe Hematology, CHU Brest, Brest, France,
  • Ranta, Dana Department of Hematology, Nancy University Hospital, Vandoeuvre-Les-Nancy, France,
  • Roy, Lydia CHU de Poitiers et INSERM CIC 802, Poitiers University Hospital, Poitiers, France,
  • Cahn, Jean-Yves Hematology, Grenoble University Hospital, Grenoble, France,
  • Harrison, Claire N Guy's and St Thomas' NHS Foundation Trust, London, United Kingdom,
  • Radia, Deepti H Guy's 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 Hospital del Mar, Hematology Department, Barcelona, Spain,
  • Cervantes, Francisco Hematology, Hospital Clinic, IDIBAPS, Barcelona, Spain,
  • Johansson, Peter Department of Medicine, Hematology and Coagulation Section, Gothenburg, Sweden,
  • Barbui, Tiziano Ospedali Riuniti di Bergamo, Bergamo, Italy,
  • Barosi, Giovanni Center for the Study of Myelofibrosis, Biotechnology Research Area, IRCCS Policlinico San Matteo Foundation, Pavia, Italy,
  • Vannucchi, Alessandro M Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy,
  • Passamonti, Francesco Department of Hematology Oncology, Fondazione IRCCS Policlinico San Matteo, University of Pavia, Pavia, Italy,
  • Andreasson, Bjorn Internal Medicine, NU Hospital Organization, Uddevalla, Sweden,
  • Ferrari, Maria Ospedali Riuniti di Bergamo, Bergamo, Italy,
  • Griesshammer, Martin Hämatologie und Onkologie, Hämostaseologie und Palliativmedizin, Johannes Wesling Klinikum Minden, Minden, Germany,
  • Rambaldi, Alessandro Adult Hematology Department, Ospedali Riuniti di Bergamo, Bergamo, Italy,
  • Samuelsson, Jan Department of Internal Medicine, Stockholm South Hospital, Stockholm, Sweden,
  • Birgegard, Gunnar Department of Medical Sciences, Uppsala University, Uppsala, Sweden,
  • Xiao, Zhijian Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China,
  • Xu, Zefeng MDS & MPN Center, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China,
  • Zhang, Yue Institute of Hematology and Blood Diseases Hospital, Tianjin, China,
  • Sun, Xiujuan MDS and MPN Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China,
  • Xu, Junqing MDS and MPN Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China,
  • Zhang, Peihong Department of Pathology, Institute of Hematology, Blood Disease Hospital, Chinese Academy of Medical Sciences, Tianjin, China,
  • Gale, Robert Peter Section of Hematology, Division of Experimental Medicine, Department of Medicine, Imperial College, London, United Kingdom,
  • Tefferi, Ayalew Division of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, MN, USA
  • Mesa, Ruben A. Mayo Clinic Arizona, Scottsdale, AZ, USA,
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Published in:
  • Blood. - American Society of Hematology. - 2013, vol. 122, no. 21, p. 4067-4067
Immunology
Cell Biology
Biochemistry
Hematology
English Abstract

Background
We have previously reported on the significant, but heterogeneous baseline MPN symptom burden among an international sample of MPN patients (including essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF)) utilizing the MPN Symptom Assessment Form (MPN-SAF) and the derivative Total Symptom Score (MPN-SAF TSS). Recent clinical trials have sought to determine optimal MPN symptom response criteria, such as absolute 10 point improvement in MPN SAF TSS for ET/PV (ELN Criteria, Barosi et. al. Blood 2013) and 50% reduction in MPN-SAF TSS for MF (IWG-MRT, Tefferi et. al. Blood 2013). We sought to determine the role of improvement in MPN-SAF TSS quartiles as potential thresholds to assess symptomatic response to therapy.


Methods
Utilizing prospectively gathered MPN-SAF TSS (Emanuel et. al. JCO 2012) in patients we assessed potential thresholds of response by evaluating quartile thresholds for severity of symptom burden. The MPN-SAF TSS was scored as the average of 10 symptoms (individual symptoms scores of 0-10, with a total score of 0 (best) to 100 (worst)). MPN-SAF TSS quartiles were identified by the percentage of scores between 0-24% (quartile 1 (Q1)), 25-49% (quartile 2 (Q2)), 50-74% (quartile 3 (Q3)), 75-100% (quartile 4 (Q4)).


Results
MPN-SAF TSS Quartiles: MPN-SAF TSS quartiles were identified among 1858 MPN patients (ET N=775, PV N=654, and MF N=423). Overall MPN-SAF TSS scores of 0 - 7 were designated as Q1, 8 - 17 as Q2, 18 - 31 as Q3, and ≥ 32 was as Q4. MPN-SAF TSS scores were significantly different between clusters (p<0.001).
Associations Between Quartiles and Demographic/ Disease Factors: As quartiles increased, the proportion of PV and ET patients diminished and MF increased (Table 1, p<0.001). Cytopenias and transfusion dependence increased in prevalence in the higher quartiles (p<0.001). A history of prior thrombosis was also significantly more prevalent in the quartiles with highest symptom burden (p<0.001). The prevalence of women was significantly higher among the more symptomatic quartiles females 48.9% Q1, 49.4% Q2, 58.4% Q3, and 60.1% Q4; p<0.001).
Associations Between Individual Symptoms and MPN-SAF TSS Quartiles: All individual symptoms measured in the MPN-SAF TSS were significantly worse in quartiles as they increased (p<0.0001).
Evaluation of Prognostic Scoring and MPN-SAF TSS Quartiles: Comparison of each patients individual risk score (IPSET, PV, DIPSS for MF) and worsening symptom quartile showed the highest correlation with MF patients (DIPSS) (Table 1). However, ET and PV risk scores were not surrogates for symptom burden by quartile.


Conclusions
Distribution of MPN patient symptomatic burden by MPN-SAF TSS quartiles provides an easy-to-calculate method to cluster and analyze MPN patients of similar burden. Although MF patients are most prevalent in the most severe quartile of MPN symptomatology it is notable that Q4 has many patients with PV and ET. Future prospective efforts are ongoing to assess the potential of using changes in quartile (i.e. improving from Q3 to Q1) as potential symptomatic response thresholds.


Disclosures:
Etienne: novartis: Consultancy, Membership on an entity’s Board of Directors or advisory committees; Bristol Myers Squibb: Consultancy, Membership on an entity’s Board of Directors or advisory committees; Pfizer: Membership on an entity’s Board of Directors or advisory committees; Ariad: Membership on an entity’s Board of Directors or advisory committees. Roy:Novartis, BMS: Honoraria. Harrison:Gilead: Honoraria, Membership on an entity’s Board of Directors or advisory committees; S Bio: Honoraria, Membership on an entity’s Board of Directors or advisory committees; Shire: Speakers Bureau; Celgene: Honoraria; YM Bioscience: Honoraria, Membership on an entity’s Board of Directors or advisory committees; Sanofi: Honoraria, Membership on an entity’s Board of Directors or advisory committees, Speakers Bureau; Novartis: Honoraria, Membership on an entity’s Board of Directors or advisory committees, Research Funding, Speakers Bureau. Vannucchi:Novartis: Honoraria, Membership on an entity’s Board of Directors or advisory committees. Birgegard:Vifor Pharma: Honoraria.
Language
  • English
Open access status
closed
Identifiers
Persistent URL
https://folia.unifr.ch/global/documents/5631
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