Conventional therapeutic options have limited impact on MPN symptoms: Insights from a prospective analysis of the MPN-SAF TSS

Scherber R., Dueck A., Kiladjian J., Slot S., Zweegman S., Te Boekhorst P., Commandeur S., Schouten H., Sackmann F., Fuentes A., Hernandez-Maraver D., Pahl H., Greiesshammer M., Stegelmann F., Doehner K., Lehmann T., Bonatz K., Reiter A., Boyer F., Etienne G.Ianotto J., Roy L., Cahn J., Harrison C., Radia D., Muxi P., Maldonado N., Besses C., Cervantes F., Johansson P., Barbui T., Barosi G., Vannucchi A., Passamonti F., Andreasson B., Ferarri M., Rambaldi A., Samuelsson J., Birgegard G., Tefferi A., Mesa R.

Research output: Chapter in Book/Report/Conference proceedingChapterAcademicpeer-review


Background. Symptom burden in MPNs is severe and a risk factor for mortality in some disease subtypes (Blood 2010;115(9):1703-8). A recent trial comparing JAK2 treatment to best available therapy revealed that patients receiving conventional MPN therapies experienced no difference or worsening of symptoms (Blood 2011;118(21):a6501). No studies have evaluated the specific associations of conventional therapies on individual MPN symptoms using a validated patient-reported measure of symptom burden. Aims. We aimed to assess associations between conventional therapies on specific MPN symptoms using the MPN-SAF TSS (Blood 2011;118(21):a3839). Methods. Patient demographics, symptom burden and disease traits including therapies were collected from MPN patients and physicians at a single time point during therapy. MPN-SAF TSS included "worst"fatigue from the BFI and MPN-SAF items of concentration, early satiety, inactivity, night sweats, itching, bone pain, abdominal discomfort, weight loss and fever. MPN-SAF TSS was calculated as the total 10-item score, reported in a range of 0 to 100 for patients completing at least 6 of the 10 items. Results. Demographics: 1433 MPN patients were prospectively enrolled, including 594 essential thrombocythemia(ET), 538 polycythemia vera(PV) and 293 myelofibrosis(MF) patients from 11 countries. Of these, 1408 patients completed at least 6 of the 10 required items. Patients were 54% female with a median age of 63.Therapies: Common therapies included hydroxyurea/hydroxycarbamide(47.7%), salicylates(43.8%), interferon/pegylated- interferon(9.4%), and phlebotomies(5.3%) with 12.6% receiving no therapy. MPN-SAF TSS variations were observed between patients receiving and not receiving each therapy, although only one reached statistical significance (Table 1). Overall Treatment Effects: When comparing the overall effects of current therapy, worst fatigue was significantly more severe among PV patients undergoing treatment as compared to patients not receiving treatment (4.5(n=497) vs. 3.4(n=30), p=0.03). Among MF patients, abdominal comfort was significantly worse among patients not receiving therapy (3.3(n=57) vs. 2.3(n=230),p=0.02) .Aspirin: ET patients receiving aspirin reported significantly less weight loss than patients not receiving salicylates (0.62(n=298) vs. 0.98(n=283),p=0.03). Additionally, aspirin among ET patients reduced pruritus (1.5(n=296) vs. 2.0(n=284),p=0.02). PV patients receiving aspirin reported more concentration difficulties than patients on a non-aspirin regimen (3.0(n=254) vs. 2.4(n=264),p=0.01). MF patients receiving aspirin reported decreased bone pain (1.5(n=58) vs. 2.4(n=228),p=0.04) and abdominal discomfort (1.9(n=58) vs. 2.7(n=229),p=0.04). Hydroxyurea/hydroxycarbamide: PV patients receiving hydroxyurea/hydroxycarbamide reported decreased itching than patients not on this therapy, although this effect was of borderline significance (2.5(n=272) vs. 3.0(n=255),p=0.05). Phlebotomies: PV patients being given phlebotomies had significantly decreased problems with concentration compared to non-phlebotomized patients (1.9(n=71) vs. 2.8(n=447),p=0.02). Interferon/pegylatedinterferon: PV patients receiving interferon therapy reported increased early satiety (3.1(n=69) vs. 2.4(n=257),p=0.04), fever (0.7(n=67) vs. 0.3(n=455), p=0.02), and MPN-SAF TSS (25.5(n=69) vs. 21.2(n=460),p=0.04) than noninterferon- receiving counterparts. Conclusions. Few significant effects in overall symptom burden were observed in patients undergoing traditional MPN therapies. Though some effects reached nominal statistical significance, a portion may be spurious given the large number of tests performed. Further studies are needed to determine whether treatments impart additional symptom burden or if patients with specific symptom characteristics are selected to receive a particular treatment. Prospective serial assessment of conventional therapy impact is ongoing as part of a new clinical trial.
Original languageEnglish
Title of host publicationHaematologica
Publication statusPublished - 2012

Publication series



  • *European
  • *hematology
  • abdominal discomfort
  • acetylsalicylic acid
  • blood
  • bone pain
  • clinical trial
  • comfort
  • fatigue
  • female
  • fever
  • human
  • interferon
  • mortality
  • myelofibrosis
  • night sweat
  • patient
  • phlebotomy
  • physician
  • polycythemia vera
  • pruritus
  • risk factor
  • salicylic acid derivative
  • satiety
  • statistical significance
  • therapy
  • thrombocythemia
  • weight reduction

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