Purpose. Polycythemia vera (PV) is amyeloproliferative neoplasm (MPN) associatedwith disabling symptoms and a heightened risk of life-threatening complications. Recent studies have demonstrated the effectiveness of JAK inhibitor therapy in patients with PV patients who have a history of prior hydroxyurea (HU) use (including resistance or intolerance), phlebotomy requirements, and palpable splenomegaly. We aimed to determine how these features contribute alone and in aggregate to the PV symptom burden. Patients and Methods Through prospective evaluation of 1,334 patients with PV who had characterized symptom burden, we assessed patient demographics, laboratory data, and the presence of splenomegaly by disease feature (ie, known HU use, known phlebotomy requirements, splenomegaly). Results. The presence of each feature in itself is associated with a moderately high symptom burden (MPN symptom assessment form [SAF] total symptom score [TSS] range, 27.7 to 29.2) that persists independent of PV risk category. In addition, symptoms incrementally increase in severity with the addition of other features. Patients with PV who had all three features (PV-HUPS) faced the highest total score (MPN-SAF TSS, 32.5) but had similar individual symptom scores to patients with known HU use (PV-HU), known phlebotomy (PV-P), and splenomegaly (PV-S). Conclusion. The results of this study suggest that patientswith PVwho have any one of the features in question (known HU use, known phlebotomy, or splenomegaly) have significant PV-associated symptoms. Furthermore, it demonstrates that many PV symptoms remain severe independent of the number of features present. Copyright © 2016 American Society of Clinical Oncology. All rights reserved.

Symptomatic profiles of patients with polycythemia vera: Implications of inadequately controlled disease

PASSAMONTI, FRANCESCO
Writing – Original Draft Preparation
;
2016-01-01

Abstract

Purpose. Polycythemia vera (PV) is amyeloproliferative neoplasm (MPN) associatedwith disabling symptoms and a heightened risk of life-threatening complications. Recent studies have demonstrated the effectiveness of JAK inhibitor therapy in patients with PV patients who have a history of prior hydroxyurea (HU) use (including resistance or intolerance), phlebotomy requirements, and palpable splenomegaly. We aimed to determine how these features contribute alone and in aggregate to the PV symptom burden. Patients and Methods Through prospective evaluation of 1,334 patients with PV who had characterized symptom burden, we assessed patient demographics, laboratory data, and the presence of splenomegaly by disease feature (ie, known HU use, known phlebotomy requirements, splenomegaly). Results. The presence of each feature in itself is associated with a moderately high symptom burden (MPN symptom assessment form [SAF] total symptom score [TSS] range, 27.7 to 29.2) that persists independent of PV risk category. In addition, symptoms incrementally increase in severity with the addition of other features. Patients with PV who had all three features (PV-HUPS) faced the highest total score (MPN-SAF TSS, 32.5) but had similar individual symptom scores to patients with known HU use (PV-HU), known phlebotomy (PV-P), and splenomegaly (PV-S). Conclusion. The results of this study suggest that patientswith PVwho have any one of the features in question (known HU use, known phlebotomy, or splenomegaly) have significant PV-associated symptoms. Furthermore, it demonstrates that many PV symptoms remain severe independent of the number of features present. Copyright © 2016 American Society of Clinical Oncology. All rights reserved.
2016
https://www.scopus.com/inward/record.uri?eid=2-s2.0-84954221953&partnerID=40&md5=61e2c40ee363e872bd3bba9a19416733
hemoglobin; hydroxyurea; antineoplastic agent; hydroxyurea; Janus kinase, abdominal discomfort; adult; anemia; bone pain; Conference Paper; controlled study; disease duration; fatigue; fever; hemoglobin blood level; human; leukocytosis; leukopenia; major clinical study; middle aged; night sweat; palpable splenomegaly; phlebotomy; polycythemia vera; priority journal; prospective study; pruritus; splenomegaly; symptom assessment; thrombocytopenia; thrombocytosis; validation study; weight reduction; aged; antagonists and inhibitors; complication; fatigue; female; fever; genetics; male; pain; palpation; phlebotomy; polycythemia vera; prognosis; pruritus; severity of illness index; splenomegaly; sweating; very elderly, Adult; Aged; Aged, 80 and over; Antineoplastic Agents; Fatigue; Female; Fever; Humans; Hydroxyurea; Janus Kinases; Male; Middle Aged; Pain; Palpation; Phlebotomy; Polycythemia Vera; Prognosis; Prospective Studies; Pruritus; Severity of Illness Index; Splenomegaly; Sweating; Weight Loss
Geyer, H.; Scherber, R.; Kosiorek, H.; Dueck, A. C.; Kiladjian, J. J.; Xiao, Z.; Slot, S.; Zweegman, S.; Sackmann, F.; Fuentes, A. K.; Hernández Maraver, D.; Döhner, K.; Harrison, C. N.; Radia, D.; Muxi, P.; Besses, C.; Cervantes, F.; Johansson, P. L.; Andreasson, B.; Rambaldi, A.; Barbui, T.; Bonatz, K.; Reiter, A.; Boyer, F.; Etienne, G.; Ianotto, J. C.; Ranta, D.; Roy, L.; Cahn, J. Y.; Maldonado, N.; Barosi, G.; Ferrari, M. L.; Gale, R. P.; Birgegard, G.; Xu, Z.; Zhang, Y.; Sun, X.; Xu, J.; Zhang, P.; Te Boekhorst, P. A. W.; Commandeur, S.; Schouten, H.; Pahl, H. L.; Griesshammer, M.; Stegelmann, F.; Lehmann, T.; Senyak, Z.; Vannucchi, A. M.; Passamonti, Francesco; Samuelsson, J.; Mesa, R. A.
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11383/2046347
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