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1. Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis (MOMENTUM): results from an international, double-blind, randomised, controlled, phase 3 study

Author

Abulafia, Adi Shacham, Al-Ali, Haifa Kathrin, Andreasson, Bjorn, Angona, Anna, Ayala, Rosa, Bang, Soo-Mee, Bank, Bruce, Barraco, Fiorenza, Beggiato, Eloise, Benghiat, Fleur Samantha, Bonifacio, MassimiliaNo, Bories, Claire, Borsaru, Gabriela, Brabrand, Mette, Braester, Andrei, Broliden, Andes, Buxhofer-Ausch, Veronika, Cambier, Nathalie, Caramella, Marianna, Carpentier, Benjamin, Cascavilla, Nicola, Castellano, Maria Giraldo, Chang, Hung, Chen, Chih-Cheng, Cheong, June-Won, Choi, Yunsuk, Choi, Philip, Corsetti, Maria Teresa, Cuadrado, Isabel Montero, Cunningham, Julia, Damaj, Gandhi Laurent, De Stefano, Valerio, Delage, Robert, Delgado, Regina Garcĺa, Diaz, Jose Miguel Torregrosa, Dombi, Péter, Dubruille, Viviane, Egyed, Miklós, El Fassi, Daniel, Elinder-Camburn, Anna, Elli, Elena Maria, Ellis, Martin, Fava, Carmen, Fazal, Salman, Fleischman, Angela, Foltz, Lynda, Fox, Laura, Gabrail, Nashat, Garcĺa-Gutiérrez, Jose Valentĺn, Gerds, Aaron, Girault, Stephane, Gisslinger, Heinz, Gluvacov, Alexandru, Goh, Yeow Tee, Göthert, Joachim, Granacher, Nikki, Grosicki, Sebastian, Gupta, Vikas, Hadjiev (Hadzhiev), Evgeni (Evgueniy), Hafraoui, Kaoutar, Hamed, Aryan, Harrison, Claire, Hasselbalch, Hans, Hauser, Hanns, Heaney, Mark, Hebart, Holger, Hernandez Rivas, Jesus Maria, Higuero Saavedra, Victor, Hillis, Christopher, Hou, Hsin-An, How, Jonathan, Huang, Daniel, Hus, Marek, Illés, Arpad, Isidori, Alessandro, Iurlo, Alessandra, Ivanov, Vadim, Johansson, Peter, Jung, Chul Won, Kiladjian, Jean-Jacques, Kirgner, Ilya, Koren-Michowitz, Maya, Koschmieder, Steffen, Kosztolanyi, Szabolcs Ors, Kreiniz, Natalia, Kuykendall, Andrew, Lambert, Jonathan, Laribi, Kamel, Lascaux, Axelle, Lavie, Noa, Lavie, David, Lazaroiu, Mihaela, Leahy, Michael, Lech-Maranda, Ewa, Lee, Sung-Eun, Lee, Won Sik, Legrand, Ollivier, Lemoli, Roberto, Liang, James, Lim, Sung-Nam, Loschi, Michael, Lucchesi, Alessandro, Macarie, Ioan, Marolleau, Jean-Pierre, Martelli, Maurizio, Mayer, Jiri, McCloskey, James, McDermott, Christopher, McLornan, Donal, McMahon, Brandon, Mehta, Priyanka, Mesa, Ruben, Mikala, Gábor, Milojkovic, Dragana, Mineur, Philippe, Mishchenko, Elena, Moon, Joon Ho, Nagy, Zsolt, Narayanan, Srinivasan, O'Connell, Casey, Ocroteala, Luminita, Oh, Stephen, Ojeda-Uribe, Mario, Ong, Kiat Hoe, Otegbeye, Folashade, Palmer, Jeanne, Pane, Fabrizio, Passamonti, Francesco, Patriarca, Andrea, Perkins, Andrew, Pietrantuono, Giuseppe, Plander, Mark, Platzbecker, Uwe, Prasad, Ritam, Prejzner, Witold, Rachow, Tobias, Radinoff, Atanas, Rejtő, László, Rinaldi, Ciro, Robak, Tadeusz, Rodriguez, Maria Angeles Fernandez, Ronson, Aaron, Ross, David, Sacha, Tomasz, Sadjadian, Parvis, Salar, Antonio, Santillana, Guillermo Sanz, Scheid, Christof, Schmidt, Aline, Severinsen, Marianne Tang, Stoeva, Vera, Szwedyk, Paweł, Tiribelli, Mario, Trautmann-Grill, Karolin, Trottier, Amy, Tzvetkov, Nikolay, van Droogenbroeck, Janusz, Vannucchi, Alessandro, Verstovsek, Srdan, Vianelli, Nicola, von Bubnoff, Nikolas, Wolf, Dominik, Woszczyk, Dariusz, Woźny, Tomasz, Wróbel, Tomasz, Xicoy, Blanca, Yeh, Su-Peng, Yoon, Sung-Soo, Gerds, Aaron T, Vannucchi, Alessandro M, Kuykendall, Andrew T, Lazaroiu, Mihaela C, Egyed, Miklos, Fox, Maria Laura, Harrison, Claire N, Klencke, Barbara J, Ro, Sunhee, Donahue, Rafe, and Kawashima, Jun

Published
2023
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2. Longitudinal minimal residual disease assessment in multiple myeloma patients in complete remission – results from the NMSG flow-MRD substudy within the EMN02/HO95 MM trial

Author

Schmitz, Alexander, Brøndum, Rasmus Froberg, Johnsen, Hans Erik, Mellqvist, Ulf-Henrik, Waage, Anders, Gimsing, Peter, op Bruinink, Davine Hofste, van der Velden, Vincent, van der Holt, Bronno, Hansson, Markus, Andersen, Niels Frost, Frølund, Ulf Christian, Helleberg, Carsten, Schjesvold, Fredrik H., Ahlberg, Lucia, Gulbrandsen, Nina, Andreasson, Bjorn, Lauri, Birgitta, Haukas, Einar, Bødker, Julie Støve, Roug, Anne Stidsholt, Bøgsted, Martin, Severinsen, Marianne T., Gregersen, Henrik, Abildgaard, Niels, Sonneveld, Pieter, and Dybkær, Karen

Published
2022
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3. Symptom burden profile in myelofibrosis patients with thrombocytopenia: Lessons and unmet needs

Author

Scotch, Allison H., Kosiorek, Heidi, Scherber, Robyn, Dueck, Amylou C., Slot, Stefanie, Zweegman, Sonja, Boekhorst, Peter A.W.te, Commandeur, Suzan, Schouten, Harry, Sackmann, Federico, Fuentes, Ana Kerguelen, Hernández-Maraver, Dolores, Pahl, Heike L., Griesshammer, Martin, Stegelmann, Frank, Döhner, Konstanze, Lehmann, Thomas, Bonatz, Karin, Reiter, Andreas, Boyer, Francoise, Etienne, Gabriel, Ianotto, Jean-Christophe, Ranta, Dana, Roy, Lydia, Cahn, Jean-Yves, Harrison, Claire N., Radia, Deepti, Muxi, Pablo, Maldonado, Norman, Besses, Carlos, Cervantes, Francisco, Johansson, Peter L., Barbui, Tiziano, Barosi, Giovanni, Vannucchi, Alessandro M., Paoli, Chiara, Passamonti, Francesco, Andreasson, Bjorn, Ferrari, Maria L., Rambaldi, Alessandro, Samuelsson, Jan, Birgegard, Gunnar, Xiao, Zhijian, Xu, Zefeng, Zhang, Yue, Sun, Xiujuan, Xu, Junqing, Kiladjian, Jean-Jacques, Zhang, Peihong, Gale, Robert Peter, Mesa, Ruben A., and Geyer, Holly L.

Published
2017
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4. Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis (MOMENTUM): results from an international, double-blind, randomised, controlled, phase 3 study

Author

Verstovsek, Srdan, primary, Gerds, Aaron T, additional, Vannucchi, Alessandro M, additional, Al-Ali, Haifa Kathrin, additional, Lavie, David, additional, Kuykendall, Andrew T, additional, Grosicki, Sebastian, additional, Iurlo, Alessandra, additional, Goh, Yeow Tee, additional, Lazaroiu, Mihaela C, additional, Egyed, Miklos, additional, Fox, Maria Laura, additional, McLornan, Donal, additional, Perkins, Andrew, additional, Yoon, Sung-Soo, additional, Gupta, Vikas, additional, Kiladjian, Jean-Jacques, additional, Granacher, Nikki, additional, Lee, Sung-Eun, additional, Ocroteala, Luminita, additional, Passamonti, Francesco, additional, Harrison, Claire N, additional, Klencke, Barbara J, additional, Ro, Sunhee, additional, Donahue, Rafe, additional, Kawashima, Jun, additional, Mesa, Ruben, additional, Abulafia, Adi Shacham, additional, Andreasson, Bjorn, additional, Angona, Anna, additional, Ayala, Rosa, additional, Bang, Soo-Mee, additional, Bank, Bruce, additional, Barraco, Fiorenza, additional, Beggiato, Eloise, additional, Benghiat, Fleur Samantha, additional, Bonifacio, MassimiliaNo, additional, Bories, Claire, additional, Borsaru, Gabriela, additional, Brabrand, Mette, additional, Braester, Andrei, additional, Broliden, Andes, additional, Buxhofer-Ausch, Veronika, additional, Cambier, Nathalie, additional, Caramella, Marianna, additional, Carpentier, Benjamin, additional, Cascavilla, Nicola, additional, Castellano, Maria Giraldo, additional, Chang, Hung, additional, Chen, Chih-Cheng, additional, Cheong, June-Won, additional, Choi, Yunsuk, additional, Choi, Philip, additional, Corsetti, Maria Teresa, additional, Cuadrado, Isabel Montero, additional, Cunningham, Julia, additional, Damaj, Gandhi Laurent, additional, De Stefano, Valerio, additional, Delage, Robert, additional, Delgado, Regina Garcĺa, additional, Diaz, Jose Miguel Torregrosa, additional, Dombi, Péter, additional, Dubruille, Viviane, additional, Egyed, Miklós, additional, El Fassi, Daniel, additional, Elinder-Camburn, Anna, additional, Elli, Elena Maria, additional, Ellis, Martin, additional, Fava, Carmen, additional, Fazal, Salman, additional, Fleischman, Angela, additional, Foltz, Lynda, additional, Fox, Laura, additional, Gabrail, Nashat, additional, Garcĺa-Gutiérrez, Jose Valentĺn, additional, Gerds, Aaron, additional, Girault, Stephane, additional, Gisslinger, Heinz, additional, Gluvacov, Alexandru, additional, Göthert, Joachim, additional, Hadjiev (Hadzhiev), Evgeni (Evgueniy), additional, Hafraoui, Kaoutar, additional, Hamed, Aryan, additional, Harrison, Claire, additional, Hasselbalch, Hans, additional, Hauser, Hanns, additional, Heaney, Mark, additional, Hebart, Holger, additional, Hernandez Rivas, Jesus Maria, additional, Higuero Saavedra, Victor, additional, Hillis, Christopher, additional, Hou, Hsin-An, additional, How, Jonathan, additional, Huang, Daniel, additional, Hus, Marek, additional, Illés, Arpad, additional, Isidori, Alessandro, additional, Ivanov, Vadim, additional, Johansson, Peter, additional, Jung, Chul Won, additional, Kirgner, Ilya, additional, Koren-Michowitz, Maya, additional, Koschmieder, Steffen, additional, Kosztolanyi, Szabolcs Ors, additional, Kreiniz, Natalia, additional, Kuykendall, Andrew, additional, Lambert, Jonathan, additional, Laribi, Kamel, additional, Lascaux, Axelle, additional, Lavie, Noa, additional, Lazaroiu, Mihaela, additional, Leahy, Michael, additional, Lech-Maranda, Ewa, additional, Lee, Won Sik, additional, Legrand, Ollivier, additional, Lemoli, Roberto, additional, Liang, James, additional, Lim, Sung-Nam, additional, Loschi, Michael, additional, Lucchesi, Alessandro, additional, Macarie, Ioan, additional, Marolleau, Jean-Pierre, additional, Martelli, Maurizio, additional, Mayer, Jiri, additional, McCloskey, James, additional, McDermott, Christopher, additional, McMahon, Brandon, additional, Mehta, Priyanka, additional, Mikala, Gábor, additional, Milojkovic, Dragana, additional, Mineur, Philippe, additional, Mishchenko, Elena, additional, Moon, Joon Ho, additional, Nagy, Zsolt, additional, Narayanan, Srinivasan, additional, O'Connell, Casey, additional, Oh, Stephen, additional, Ojeda-Uribe, Mario, additional, Ong, Kiat Hoe, additional, Otegbeye, Folashade, additional, Palmer, Jeanne, additional, Pane, Fabrizio, additional, Patriarca, Andrea, additional, Pietrantuono, Giuseppe, additional, Plander, Mark, additional, Platzbecker, Uwe, additional, Prasad, Ritam, additional, Prejzner, Witold, additional, Rachow, Tobias, additional, Radinoff, Atanas, additional, Rejtő, László, additional, Rinaldi, Ciro, additional, Robak, Tadeusz, additional, Rodriguez, Maria Angeles Fernandez, additional, Ronson, Aaron, additional, Ross, David, additional, Sacha, Tomasz, additional, Sadjadian, Parvis, additional, Salar, Antonio, additional, Santillana, Guillermo Sanz, additional, Scheid, Christof, additional, Schmidt, Aline, additional, Severinsen, Marianne Tang, additional, Stoeva, Vera, additional, Szwedyk, Paweł, additional, Tiribelli, Mario, additional, Trautmann-Grill, Karolin, additional, Trottier, Amy, additional, Tzvetkov, Nikolay, additional, van Droogenbroeck, Janusz, additional, Vannucchi, Alessandro, additional, Verstovsek, Srdan, additional, Vianelli, Nicola, additional, von Bubnoff, Nikolas, additional, Wolf, Dominik, additional, Woszczyk, Dariusz, additional, Woźny, Tomasz, additional, Wróbel, Tomasz, additional, Xicoy, Blanca, additional, and Yeh, Su-Peng, additional

Published
2023
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5. Circulating YKL-40 in patients with essential thrombocythemia and polycythemia vera treated with the novel histone deacetylase inhibitor vorinostat

Author

Andersen, Christen Lykkegaard, Bjørn, Mads Emil, McMullin, Mary Frances, Harrison, Claire, Samuelsson, Jan, Ejerblad, Elisabeth, Zweegman, Sonja, Fernandes, Savio, Bareford, David, Knapper, Steven, Löfvenberg, Eva, Linder, Olle, Andreasson, Bjørn, Ahlstrand, Erik, Jensen, Morten Krogh, Bjerrum, Ole Weis, Vestergaard, Hanne, Larsen, Herdis, Klausen, Tobias Wirenfeldt, Mourits-Andersen, Torben, Skov, Vibe, Thomassen, Mads, Kruse, Torben, Grønbæk, Kirsten, and Hasselbalch, Hans Carl

Published
2014
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6. Survival and risk of vascular complications in myelofibrosis-A population-based study from the Swedish MPN group

Author

Lindgren, Marie, Andreasson, Bjorn, Samuelsson, Jan, Pettersson, Helna, Enblom-Larsson, Anneli, Ravn-Landtblom, Anna, Scheding, Stefan, Bentham, Carolina, Ahlstrand, Erik, Lindgren, Marie, Andreasson, Bjorn, Samuelsson, Jan, Pettersson, Helna, Enblom-Larsson, Anneli, Ravn-Landtblom, Anna, Scheding, Stefan, Bentham, Carolina, and Ahlstrand, Erik

Abstract

Objective To gain knowledge of underlying risk factors for vascular complications and their impact on life expectancy in myelofibrosis. Methods From a cohort of 392 myelofibrosis patients registered in the Swedish MPN registry 58 patients with vascular complications during follow-up were identified. Patients with vascular complications were compared with both 1:1 matched controls and the entire myelofibrosis cohort to explore potential risk factors for vascular complications and their impact on survival. Results Incidence of vascular complications was 2.8 events per 100 patient-years and the majority of complications were thrombotic. Patients with complications were significantly older and had lower hemoglobin when compared to the entire cohort. In the case-control analysis, no significant risk factor differences were observed. The major cause of death was vascular complications and median survival was significantly impaired in patients with vascular complications (48 months) compared to controls (92 months). Inferior survival in patients with vascular complications was found to be dependent on IPSS risk category in a Cox regression model. Conclusion Vascular complications have a considerable impact on survival in MF. At diagnosis, risk assessment by IPSS does not only predict survival but is also associated with the risk of vascular complications.

Published
2022
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7. Additional file 1 of Longitudinal minimal residual disease assessment in multiple myeloma patients in complete remission ��� results from the NMSG flow-MRD substudy within the EMN02/HO95 MM trial

Author

Schmitz, Alexander, Br��ndum, Rasmus Froberg, Johnsen, Hans Erik, Mellqvist, Ulf-Henrik, Waage, Anders, Gimsing, Peter, op Bruinink, Davine Hofste, van der Velden, Vincent, van der Holt, Bronno, Hansson, Markus, Andersen, Niels Frost, Fr��lund, Ulf Christian, Helleberg, Carsten, Schjesvold, Fredrik H., Ahlberg, Lucia, Gulbrandsen, Nina, Andreasson, Bjorn, Lauri, Birgitta, Haukas, Einar, B��dker, Julie St��ve, Roug, Anne Stidsholt, B��gsted, Martin, Severinsen, Marianne T., Gregersen, Henrik, Abildgaard, Niels, Sonneveld, Pieter, and Dybk��r, Karen

Subjects
Data_FILES
Abstract

Additional file 1.

Published
2022
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8. Impact of ruxolitinib on survival of patients with myelofibrosis in the real world: update of the ERNEST Study

Author

Guglielmelli, Paola, Ghirardi, Arianna, Carobbio, Alessandra, Masciulli, Arianna, Maccari, Chiara, Mora, Barbara, Rumi, Elisa, Triguero, Ana, Finazzi, Maria Chiara, Pettersson, Helna, Paoli, Chiara, Mannelli, Francesco, Vanni, Daniele, Rambaldi, Alessandro, Passamonti, Francesco, Alvarez-Larràn, Alberto, Andreasson, Bjorn, Vannucchi, Alessandro M., and Barbui, Tiziano

Published
2022
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9. The Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF): International Prospective Validation and Reliability Trial in 402 patients

Author

Scherber, Robyn, Dueck, Amylou C., Johansson, Peter, Barbui, Tiziano, Barosi, Giovanni, Vannucchi, Alessandro M., Passamonti, Francesco, Andreasson, Bjorn, Ferarri, Maria L., Rambaldi, Alessandro, Samuelsson, Jan, Birgegard, Gunnar, Tefferi, Ayalew, Harrison, Claire N., Radia, Deepti, and Mesa, Ruben A.

Published
2011
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11. Long-Term Safety of Momelotinib in JAK Inhibitor-Naive and Previously JAK Inhibitor-Treated Intermediate-/High-Risk Myelofibrosis Patients

Author

Harrison, Claire, Andreasson, Bjorn, Cambier, Nathalie, Gerds, Aaron, Grosicki, Sebastian, Illes, Arpad, Kiladjian, Jean-Jacques, Lavie, David, Mesa, Ruben, Palmer, Jeanne, Passamonti, Francesco, Perkins, Andrew, Sacha, Tomasz, Scheid, Christof, Todorieva-Todorova, Doroteya, Vannucchi, Alessandro, Wozny, Tomasz, Klencke, Barbara, Kowalski, Mark, Verstovsek, Srdan, Harrison, Claire, Andreasson, Bjorn, Cambier, Nathalie, Gerds, Aaron, Grosicki, Sebastian, Illes, Arpad, Kiladjian, Jean-Jacques, Lavie, David, Mesa, Ruben, Palmer, Jeanne, Passamonti, Francesco, Perkins, Andrew, Sacha, Tomasz, Scheid, Christof, Todorieva-Todorova, Doroteya, Vannucchi, Alessandro, Wozny, Tomasz, Klencke, Barbara, Kowalski, Mark, and Verstovsek, Srdan

Published
2020

12. Highly reduced survival in essential thrombocythemia and polycythemia vera patients with vascular complications during follow-up

Author

Ahlstrand, Erik, Samuelsson, Jan, Lindgren, Marie, Pettersson, Helna, Liljeholm, Maria, Ravn-Landtblom, Anna, Scheding, Stefan, Andreasson, Bjorn, Ahlstrand, Erik, Samuelsson, Jan, Lindgren, Marie, Pettersson, Helna, Liljeholm, Maria, Ravn-Landtblom, Anna, Scheding, Stefan, and Andreasson, Bjorn

Abstract

Objective To explore the relative importance of risk factors, treatments, and blood counts for the occurrence of vascular complications and their impact on life expectancy in essential thrombocythemia (ET) and polycythemia vera (PV). Methods Nested case-control study within the Swedish MPN registry. From a cohort of 922 ET patients and 763 PV patients, 71 ET and 81 PV cases with vascular complications were compared with matched controls. Results Incidence of vascular complications was 2.0 and 3.4 events per 100 patient-years in ET and PV, respectively. At diagnosis, no significant risk factor differences were observed between cases and controls in neither of the diseases. At the time of vascular event, ET complication cases did not differ significantly from controls but in PV, cases had significantly higher WBCs and were to a lesser extent treated with anti-thrombotic and cytoreductive therapy. Life expectancy was significantly decreased in both ET and PV cases compared with controls. Conclusions The risk of vascular complications is high in both ET and PV, and these complications have a considerable impact on life expectancy. The protective effect of anti-thrombotic and cytoreductive therapy for vascular complications in PV underscores the importance of avoiding undertreatment., Funding Agencies|Regional FOU Vastra Gotaland County

Published
2020
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13. A Noninterventional, Observational, European Post-Authorization Safety Study of Patients With Relapsed/Refractory Multiple Myeloma Treated With Lenalidomide

Author

MS Hematologie, Infection & Immunity, Regenerative Medicine and Stem Cells, Cancer, Gamberi, Barbara, Berthou, Christian, Hernandez, Miguel, Semenzato, Gianpietro, Tholouli, Eleni, Hájek, Roman, Caers, Jo, Dimopoulos, Meletios, Minnema, Monique C, Andreasson, Bjorn, Parreira, Joana, Crotty, Gerard, Remes, Kari, Kueenburg, Elisabeth, Rosettani, Barbara, Di Micco, Antonia, Peters, Sarah, Bacon, Pamela, Blau, Igor Wolfgang, MS Hematologie, Infection & Immunity, Regenerative Medicine and Stem Cells, Cancer, Gamberi, Barbara, Berthou, Christian, Hernandez, Miguel, Semenzato, Gianpietro, Tholouli, Eleni, Hájek, Roman, Caers, Jo, Dimopoulos, Meletios, Minnema, Monique C, Andreasson, Bjorn, Parreira, Joana, Crotty, Gerard, Remes, Kari, Kueenburg, Elisabeth, Rosettani, Barbara, Di Micco, Antonia, Peters, Sarah, Bacon, Pamela, and Blau, Igor Wolfgang

Published
2020

14. A Noninterventional, Observational, European Post-Authorization Safety Study of Patients With Relapsed/Refractory Multiple Myeloma Treated With Lenalidomide

Author

Gamberi, Barbara, primary, Berthou, Christian, additional, Hernandez, Miguel, additional, Semenzato, Gianpietro, additional, Tholouli, Eleni, additional, Hájek, Roman, additional, Caers, Jo, additional, Dimopoulos, Meletios, additional, Minnema, Monique C., additional, Andreasson, Bjorn, additional, Parreira, Joana, additional, Crotty, Gerard, additional, Remes, Kari, additional, Kueenburg, Elisabeth, additional, Rosettani, Barbara, additional, Di Micco, Antonia, additional, Peters, Sarah, additional, Bacon, Pamela, additional, and Blau, Igor Wolfgang, additional

Published
2020
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15. Inflammatory functional iron deficiency common in myelofibrosis, contributes to anaemia and impairs quality of life. From the Nordic MPN study Group

Author

Birgegard, Gunnar, Samuelsson, Jan, Ahlstrand, Erik, Ejerblad, Elisabeth, Enevold, Christian, Ghanima, Waleed, Hasselbalch, Hans, Nielsen, Claus H., Knutsen, Havar, Pedersen, Ole B., Sorensen, Anders, Andreasson, Bjorn, Birgegard, Gunnar, Samuelsson, Jan, Ahlstrand, Erik, Ejerblad, Elisabeth, Enevold, Christian, Ghanima, Waleed, Hasselbalch, Hans, Nielsen, Claus H., Knutsen, Havar, Pedersen, Ole B., Sorensen, Anders, and Andreasson, Bjorn

Abstract

Objectives The study investigates the hypothesis that inflammation in myelofibrosis (MF) like in myeloma and lymphoma, may disturb iron distribution and contribute to anaemia. Methods A cross-sectional study of 80 MF and 23 ET patients was performed. Results About 35% of anaemic MF patients had functional iron deficiency (FID) with transferrin saturation amp;lt;20 and normal or elevated S-ferritin (amp;lt;500 mu g/L). In ET, FID was rare. In MF patients with FID, 70.6% were anaemic, vs 29.4% in patients without FID (P = 0.03). Hepcidin was significantly higher in MF patients with anaemia, including transfusion-dependent patients, 50.6 vs 24.4 mu g/L (P = 0.01). There was a significant negative correlation between Hb and inflammatory markers in all MF patients: IL-2, IL-6 and TNF-alpha, (P amp;lt; 0.01-0.03), LD (P = 0.004) and hepcidin (P = 0.03). These correlations were also seen in the subgroup of anaemic MF patients (Table ). Tsat correlated negatively with CRP (P amp;lt; 0.001). Symptom burden was heavier in MF patients with FID, and MPN-SAF quality of life scores correlated with IL-6 and CRP. Conclusions The inflammatory state of MF disturbs iron turnover, FID is common and contributes to anaemia development and impairment of QoL. Anaemic MF patients should be screened for FID., Funding Agencies|Nordic Study Group for Myeloproliferative Neoplasms (NMPN)

Published
2019
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16. Myeloproliferative Neoplasm Quality of Life (MPN-QOL) Study Group: MPN Experimental Assessment of Symptoms By Utilizing Repetitive Evaluation (MEASURE) Trial

Author

Gathany, Allison, primary, Scherber, Robyn M., additional, Girardo, Marlene, additional, Kosiorek, Heidi E., additional, Dueck, Amylou C., additional, Geyer, Holly, additional, Fauble, Veena D. S., additional, Vannucchi, Alessandro M., additional, Fuentes, Ana Kerguelen, additional, Andersen, Christen Lykkegaard, additional, Ejerblad, Elisabeth, additional, Birgegard, Gunnar, additional, Johansson, Peter L., additional, Andreasson, Bjorn, additional, Ross, David M, additional, Sackman, Federico, additional, Demory, Jean-Loup, additional, Tam, Constantine S., additional, Michiels, Jan J., additional, Radia, Deepti, additional, Ewing, Joanne, additional, Wadelin, Frances, additional, Lown, Robert N., additional, McMullin, Mary Frances, additional, Swe, Wunna, additional, Cullis, Jonathan O., additional, Sargant, Nigel, additional, Moreton, Paul, additional, Cargo, Catherine, additional, Sekhar, Mallika, additional, Shekouhi, Satareh, additional, and Mesa, Ruben A., additional

Published
2018
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17. Associations between gender, disease features and symptom burden in patients with myeloproliferative neoplasms : an analysis by the MPN QOL International Working Group

Author

Geyer, Holly L., Kosiorek, Heidi, Dueck, Amylou C., Scherber, Robyn, Slot, Stefanie, Zweegman, Sonja, te Boekhorst, Peter A. W., Senyak, Zhenya, Schouten, Harry C., Sackmann, Federico, Kerguelen Fuentes, Ana, Hernandez-Maraver, Dolores, Pahl, Heike L., Griesshammer, Martin, Stegelmann, Frank, Doehner, Konstanze, Lehmann, Thomas, Bonatz, Karin, Reiter, Andreas, Boyer, Francoise, Etienne, Gabriel, Ianotto, Jean-Christophe, Ranta, Dana, Roy, Lydia, Cahn, Jean-Yves, Harrison, Claire N., Radia, Deepti, Muxi, Pablo, Maldonado, Norman, Besses, Carlos, Cervantes, Francisco, Johansson, Peter L., Barbui, Tiziano, Barosi, Giovanni, Vannucchi, Alessandro M., Paoli, Chiara, Passamonti, Francesco, Andreasson, Bjorn, Ferrari, Maria L., Rambaldi, Alessandro, Samuelsson, Jan, Cannon, Keith, Birgegård, Gunnar, Xiao, Zhijian, Xu, Zefeng, Zhang, Yue, Sun, Xiujuan, Xu, Junqing, Kiladjian, Jean-Jacques, Zhang, Peihong, Gale, Robert Peter, Mesa, Ruben A., Geyer, Holly L., Kosiorek, Heidi, Dueck, Amylou C., Scherber, Robyn, Slot, Stefanie, Zweegman, Sonja, te Boekhorst, Peter A. W., Senyak, Zhenya, Schouten, Harry C., Sackmann, Federico, Kerguelen Fuentes, Ana, Hernandez-Maraver, Dolores, Pahl, Heike L., Griesshammer, Martin, Stegelmann, Frank, Doehner, Konstanze, Lehmann, Thomas, Bonatz, Karin, Reiter, Andreas, Boyer, Francoise, Etienne, Gabriel, Ianotto, Jean-Christophe, Ranta, Dana, Roy, Lydia, Cahn, Jean-Yves, Harrison, Claire N., Radia, Deepti, Muxi, Pablo, Maldonado, Norman, Besses, Carlos, Cervantes, Francisco, Johansson, Peter L., Barbui, Tiziano, Barosi, Giovanni, Vannucchi, Alessandro M., Paoli, Chiara, Passamonti, Francesco, Andreasson, Bjorn, Ferrari, Maria L., Rambaldi, Alessandro, Samuelsson, Jan, Cannon, Keith, Birgegård, Gunnar, Xiao, Zhijian, Xu, Zefeng, Zhang, Yue, Sun, Xiujuan, Xu, Junqing, Kiladjian, Jean-Jacques, Zhang, Peihong, Gale, Robert Peter, and Mesa, Ruben A.

Abstract

The myeloproliferative neoplasms, including polycythemia vera, essential thrombocythemia and myelofibrosis, are distinguished by their debilitating symptom profiles, life-threatening complications and profound impact on quality of life. The role gender plays in the symptomatology of myeloproliferative neoplasms remains under-investigated. In this study we evaluated how gender relates to patients' characteristics, disease complications and overall symptom expression. A total of 2,006 patients (polycythemia vera=711, essential thrombocythemia=830, myelofibrosis=460, unknown=5) were prospectively evaluated, with patients completing the Myeloproliferative Neoplasm-Symptom Assessment Form and Brief Fatigue Inventory Patient Reported Outcome tools. Information on the individual patients' characteristics, disease complications and laboratory data was collected. Consistent with known literature, most female patients were more likely to have essential thrombocythemia (48.6% versus 33.0%; P<0.001) and most male patients were more likely to have polycythemia vera (41.8% versus 30.3%; P<0.001). The rate of thrombocytopenia was higher among males than females (13.9% versus 8.2%; P<0.001) and males also had greater red-blood cell transfusion requirements (7.3% versus 4.9%; P=0.02) with shorter mean disease duration (6.4 versus 7.2 years, P=0.03). Despite there being no statistical differences in risk scores, receipt of most therapies or prior complications (hemorrhage, thrombosis), females had more severe and more frequent symptoms for most individual symptoms, along with overall total symptom score (22.8 versus 20.3; P<0.001). Females had particularly high scores for abdominal-related symptoms (abdominal pain/discomfort) and microvascular symptoms (headache, fatigue, insomnia, concentration difficulties, dizziness; all P<0.01). Despite complaining of more severe symptom burden, females had similar quality of life scores to those of males. The results of this study

Published
2017
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18. Symptom burden profile in myelofibrosis patients with thrombocytopenia : Lessons and unmet needs

Author

Scotch, Allison H, Kosiorek, Heidi, Scherber, Robyn, Dueck, Amylou C, Slot, Stefanie, Zweegman, Sonja, Boekhorst, Peter A W Te, Commandeur, Suzan, Schouten, Harry, Sackmann, Federico, Fuentes, Ana Kerguelen, Hernández-Maraver, Dolores, Pahl, Heike L, Griesshammer, Martin, Stegelmann, Frank, Döhner, Konstanze, Lehmann, Thomas, Bonatz, Karin, Reiter, Andreas, Boyer, Francoise, Etienne, Gabriel, Ianotto, Jean-Christophe, Ranta, Dana, Roy, Lydia, Cahn, Jean-Yves, Harrison, Claire N, Radia, Deepti, Muxi, Pablo, Maldonado, Norman, Besses, Carlos, Cervantes, Francisco, Johansson, Peter L, Barbui, Tiziano, Barosi, Giovanni, Vannucchi, Alessandro M, Paoli, Chiara, Passamonti, Francesco, Andreasson, Bjorn, Ferrari, Maria L, Rambaldi, Alessandro, Samuelsson, Jan, Birgegård, Gunnar, Xiao, Zhijian, Xu, Zefeng, Zhang, Yue, Sun, Xiujuan, Xu, Junqing, Kiladjian, Jean-Jacques, Zhang, Peihong, Gale, Robert Peter, Mesa, Ruben A, Geyer, Holly L, Scotch, Allison H, Kosiorek, Heidi, Scherber, Robyn, Dueck, Amylou C, Slot, Stefanie, Zweegman, Sonja, Boekhorst, Peter A W Te, Commandeur, Suzan, Schouten, Harry, Sackmann, Federico, Fuentes, Ana Kerguelen, Hernández-Maraver, Dolores, Pahl, Heike L, Griesshammer, Martin, Stegelmann, Frank, Döhner, Konstanze, Lehmann, Thomas, Bonatz, Karin, Reiter, Andreas, Boyer, Francoise, Etienne, Gabriel, Ianotto, Jean-Christophe, Ranta, Dana, Roy, Lydia, Cahn, Jean-Yves, Harrison, Claire N, Radia, Deepti, Muxi, Pablo, Maldonado, Norman, Besses, Carlos, Cervantes, Francisco, Johansson, Peter L, Barbui, Tiziano, Barosi, Giovanni, Vannucchi, Alessandro M, Paoli, Chiara, Passamonti, Francesco, Andreasson, Bjorn, Ferrari, Maria L, Rambaldi, Alessandro, Samuelsson, Jan, Birgegård, Gunnar, Xiao, Zhijian, Xu, Zefeng, Zhang, Yue, Sun, Xiujuan, Xu, Junqing, Kiladjian, Jean-Jacques, Zhang, Peihong, Gale, Robert Peter, Mesa, Ruben A, and Geyer, Holly L

Abstract

Myelofibrosis is a myeloproliferative neoplasm associated with progressive cytopenias and high symptom burden. MF patients with thrombocytopenia have poor prognosis but the presence of thrombocytopenia frequently precludes the use of JAK2 inhibitors. In this study, we assessed quality of life and symptom burden in 418 MF patients with (n = 89) and without (n = 329) thrombocytopenia using prospective data from the MPN-QOL study group database, including the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) and Total Symptom Score (MPN10). Thrombocytopenia, defined as platelet count < 100 x10(9)/L (moderate 51-100 x 10(9)/L; severe <= 50 x10(9)/L), was associated with anemia (76% vs. 45%, p < 0.001), leukopenia (29% vs. 11%, p < 0.001), and need for red blood cell transfusion (35% vs. 19%, p = 0.002). Thrombocytopenic patients had more fatigue, early satiety, inactivity, dizziness, sad mood, cough, night sweats, itching, fever, and weight loss; total symptom scores were also higher (33 vs. 24, p < 0.001). Patients with severe thrombocytopenia were more likely to have anemia (86% vs. 67%, p = 0.04), leukopenia (40% vs. 20%, p = 0.04), and transfusion requirements (51% vs. 20%, p = 0.002) but few differences in symptoms when compared to patients with moderate thrombocytopenia. These results suggest that MF patients with thrombocytopenia experience greater symptomatic burden than MF patients without thrombocytopenia and may benefit from additional therapies.

Published
2017
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19. Symptomatic profiles of 1334 polycythemia vera patients: implications of inadequately controlled disease

Author

Geyer, Holly L., Scherber, Robyn, Kosiorek, Heidi, Dueck, Amylou C., Kiladjian, Jean-Jacques, Xiao, Zhijian, Slot, Stefanie, Zweegman, Sonja, Sackmann, Federico, Kerguelen Fuentes, Ana, Hernández-Maraver, Dolores, Döhner, Konstanze, Harrison, Claire N., Radia, Deepti, Muxi, Pablo, Besses, Carlos, Cervantes Requena, F., Johansson, Peter L., Andreasson, Bjorn, Rambaldi, Alessandro, Barbui, Tiziano, Bonatz, Karin, Reiter, Andreas, Boyer, Francoise, Etienne, Gabriel, Ianotto, Jean-Christophe, Ranta, Dana, Roy, Lydia, Cahn, Jean-Yves, Maldonado, Norman, Barosi, Giovanni, Ferrari, Maria L., Gale, Robert Peter, Birgegard, Gunnar, Xu, Zefeng, Zhang, Yue, Sun, Xiujuan, Xu, Junqing, Zhang, Peihong, Te Boekhorst, Peter A., Commandeur, Suzan, Schouten, Harry C., Pahl, Heike L., Griesshammer, Martin, Stegelmann, Frank, Lehmann, Thomas, Senyak, Zhenya, Vannucchi, Alessandro M., Passamonti, Francesco, Samuelsson, Jan, Mesa, Ruben A., and Universitat de Barcelona

Subjects
Oncologia, Oncology, Medul·la òssia, Malalties hematològiques, Hematologic diseases, Bone marrow, Hematology, Hematologia
Abstract

Purpose Polycythemia vera (PV) is a myeloproliferative neoplasm (MPN) associated with 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 patients with PV who 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.

Published
2016

20. Myeloproliferative Neoplasm (MPN) Symptom Assessment Form Total Symptom Score: Prospective International Assessment of an Abbreviated Symptom Burden Scoring System Among Patients With MPNs

Author

Emanuel, Robyn M, Dueck, Amylou C, Geyer, Holly L, Kiladjian, Jean-Jacques, Slot, Stefanie, Zweegman, Sonja, Te Boekhorst, Peter A W, Commandeur, Suzan, Schouten, Harry C, Sackmann, Federico, Kerguelen Fuentes, Ana, Hernández-Maraver, Dolores, Pahl, Heike L, Griesshammer, Martin, Stegelmann, Frank, Doehner, Konstanze, Lehmann, Thomas, Bonatz, Karin, Reiter, Andreas, Boyer, Francoise, Etienne, Gabriel, Ianotto, Jean-Christophe, Ranta, Dana, Roy, Lydia, Cahn, Jean-Yves, Harrison, Claire N, Radia, Deepti, Muxi, Pablo, Maldonado, Norman, Besses, Carlos, Cervantes, Francisco, Johansson, Peter L, Barbui, Tiziano, Barosi, Giovanni, Vannucchi, Alessandro M, Passamonti, Francesco, Andreasson, Bjorn, Ferrari, Maria L, Ferarri, Maria L, Rambaldi, Alessandro, Samuelsson, Jan, Birgegard, Gunnar, Tefferi, Ayalew, Mesa, Ruben A, Interne Geneeskunde, RS: GROW - School for Oncology and Reproduction, Service d'hématologie clinique [Avicenne], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Université Paris 13 (UP13)-Hôpital Avicenne [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Paleontology section, Transvaal Museum, Département d'Hématologie, Institut de Cancéro-hématologie, Hôpital Morvan [Brest], Service d'Hematologie et Médecine Interne, Centre Hospitalier Régional Universitaire de Nancy (CHRU Nancy), CIC - Poitiers, Université de Poitiers-Centre hospitalier universitaire de Poitiers (CHU Poitiers)-Direction Générale de l'Organisation des Soins (DGOS)-Institut National de la Santé et de la Recherche Médicale (INSERM), TheREx, Techniques de l'Ingénierie Médicale et de la Complexité - Informatique, Mathématiques et Applications, Grenoble - UMR 5525 (TIMC-IMAG), VetAgro Sup - Institut national d'enseignement supérieur et de recherche en alimentation, santé animale, sciences agronomiques et de l'environnement (VAS)-Institut polytechnique de Grenoble - Grenoble Institute of Technology (Grenoble INP )-Centre National de la Recherche Scientifique (CNRS)-Université Joseph Fourier - Grenoble 1 (UJF)-VetAgro Sup - Institut national d'enseignement supérieur et de recherche en alimentation, santé animale, sciences agronomiques et de l'environnement (VAS)-Institut polytechnique de Grenoble - Grenoble Institute of Technology (Grenoble INP )-Centre National de la Recherche Scientifique (CNRS)-Université Joseph Fourier - Grenoble 1 (UJF), Hematology, Ospedali Riuniti, Department of Oncology and Hematology, Università degli Studi di Modena e Reggio Emilia (UNIMORE), Department of Haematology, Division of Medicine, Institution for Medical Sciences, University Hospital, and CCA - Innovative therapy

Subjects
Male, Cancer Research, Disease, Severity of Illness Index, 0302 clinical medicine, fluids and secretions, MESH: Aged, 80 and over, Quality of life, Weight loss, Sickness Impact Profile, Surveys and Questionnaires, Prospective Studies, Young adult, Prospective cohort study, Aged, 80 and over, MESH: Aged, MESH: Middle Aged, food and beverages, ORIGINAL REPORTS, Middle Aged, 3. Good health, MESH: Sickness Impact Profile, Oncology, MESH: Young Adult, 030220 oncology & carcinogenesis, Female, medicine.symptom, MESH: Myeloproliferative Disorders, Adult, medicine.medical_specialty, myelofibrosis, [SDV.CAN]Life Sciences [q-bio]/Cancer, 03 medical and health sciences, Young Adult, Internal medicine, MESH: Severity of Illness Index, Severity of illness, medicine, otorhinolaryngologic diseases, Humans, Bone pain, Myeloproliferative neoplasm, Aged, Myeloproliferative Disorders, MESH: Humans, business.industry, MESH: Questionnaires, MESH: Quality of Life, MESH: Adult, medicine.disease, equipment and supplies, bacterial infections and mycoses, MESH: Male, MESH: Prospective Studies, Surgery, carbohydrates (lipids), Quality of Life, business, MESH: Female, 030215 immunology
Abstract

Purpose 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. Patients and 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. Conclusion 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.

Published
2012

21. European Post-Approval Safety Study (EU Pass) of Relapsed/Refractory Multiple Myeloma (RRMM): Safety, Including Second Primary Malignancies, in a Large Cohort of Patients (Pts) Treated With Lenalidomide (LEN), Thalidomide (THAL), and Bortezomib (BORT)

Author

Gamberi, Barbara, primary, Hernandez, Miguel T., additional, Berthou, Christian, additional, Tholouli, Eleni, additional, Zamagni, Elena, additional, Hajek, Roman, additional, Minnema, Monique, additional, Dimopoulos, Meletios Athanasios, additional, Caers, Jo, additional, Andersen, Niels Frost, additional, Andreasson, Bjorn, additional, Waage, Anders, additional, Crotty, Gerard, additional, Kueenburg, Elisabeth, additional, Rosettani, Barbara, additional, Di Micco, Antonio, additional, Bacon, Pamela, additional, and Blau, Igor Wolfgang, additional

Published
2017
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22. European Post-Approval Safety Study (PASS) of Relapsed/Refractory Multiple Myeloma (RRMM): Safety, Including SPM, in a Large Cohort of Patients Treated with Lenalidomide, Thalidomide, and Bortezomib

Author

Gamberi, Barbara, primary, Hernandez, Miguel, additional, Berthou, Christian, additional, Tholouli, Eleni, additional, Zamagni, Elena, additional, Hájek, Roman, additional, Minnema, Monique, additional, Dimopoulos, Meletios A, additional, Caers, Jo, additional, Frost Andersen, Niels, additional, Andreasson, Bjorn, additional, Waage, Anders, additional, Crotty, Gerard, additional, Kueenburg, Elisabeth, additional, Rosettani, Barbara, additional, Di Micco, Antonia, additional, Bacon, Pamela, additional, and Blau, Igor, additional

Published
2016
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23. Myeloproliferative Neoplasm Quality of Life (MPN-QOL) Study Group: Interim Results from the MPN Experimental Assessment of Symptoms By Utilizing Repetitive Evaluation (MEASURE) Trial

Author

Scotch, Allison H, primary, Scherber, Robyn M., additional, Zhang, Nan, additional, Kosiorek, Heidi E., additional, Dueck, Amylou C., additional, Geyer, Holly, additional, Fauble, Veena, additional, Vannucchi, Alessandro M., additional, Kerguelen Fuentes, Ana, additional, Andersen, Christen Lykkegaard, additional, Ejerblad, Elisabeth, additional, Birgegard, Gunnar, additional, Johansson, Peter L., additional, Andreasson, Bjorn, additional, Ross, David M., additional, Sackmann, Federico, additional, Demory, Jean-Loup, additional, Tam, Constantine S., additional, Michiels, Jan J., additional, Radia, Deepti, additional, Mahalakshmi, Mohan, additional, Jennings, Daniel, additional, Ewing, Joanne C, additional, Wadelin, Frances, additional, Lown, Robert N, additional, Drew, Victoria, additional, Hudson, John, additional, McMullin, Mary Frances, additional, Swe, Wunna, additional, Cullis, Jonathan O., additional, Sargant, Nigel, additional, Moreton, Paul, additional, Kilner, Mari Frances, additional, Cargo, Catherine, additional, Sekhar, Mallika, additional, Shekouhi, Satareh, additional, and Mesa, Ruben A., additional

Published
2016
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24. Symptom Burden As Primary Driver for Therapy in Patients with Myelofibrosis: An Analysis By MPN International Quality of Life Study Group

Author

Scherber, Robyn M., primary, Geyer, Holly, additional, Dueck, Amylou Constance, additional, Kosiorek, Heidi E., additional, Kiladjian, Jean-Jacques, additional, Slot, Stefanie, additional, Zweegman, Sonja, additional, Te Boekhorst, Peter, additional, Commandeur, Suzan, additional, Schouten, Harry C., additional, Sackmann, Federico, additional, Fuentes, Ana Kerguelen, additional, Hernandez-Maraver, Dolores, additional, Pahl, Heike L, additional, Griesshammer, Martin, additional, Stegelmann, Frank, additional, Doehner, Konstanze, additional, Lehmann, Thomas, additional, Bonatz, Karen, additional, Reiter, Andreas, additional, Boyer, Francoise, additional, Etienne, Gabriel, additional, Ianotto, Jean Christophe, additional, Ranta, Dana, additional, Roy, Lydia, additional, Cahn, Jean-Yves, additional, Harrison, Claire N., additional, Radia, Deepti, additional, Muxi, Pablo J., additional, Maldonado, Norman I, additional, Besses, Carlos, additional, Cervantes, Francisco, additional, Johansson, Peter L., additional, Barbui, Tiziano, additional, Barosi, Giovanni, additional, Vannucchi, Alessandro M., additional, Andreasson, Bjorn, additional, Ferarri, Maria, additional, Rambaldi, Alessandro, additional, Samuelsson, Jan, additional, Birgegard, Gunnar, additional, Cannon, Keith, additional, Sun, Xuijuan, additional, Xu, Junqing, additional, Zhang, Peihong, additional, Xu, Zefeng, additional, and Mesa, Ruben A., additional

Published
2016
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25. Consideration of Symptom Burden Based Treatment in PV and ET Patients: An Analysis By MPN International Quality of Life Study Group

Author

Scherber, Robyn M., primary, Geyer, Holly, additional, Kosiorek, Heidi E., additional, Dueck, Amylou C., additional, Kiladjian, Jean-Jacques, additional, Slot, Stefanie, additional, Zweegman, Sonja, additional, Te Boekhorst, Peter, additional, Commandeur, Suzan, additional, Schouten, Harry C., additional, Sackmann, Federico, additional, Fuentes, Ana Kerguelen, additional, Hernandez-Maraver, Dolores, additional, Pahl, Heike L, additional, Griesshammer, Martin, additional, Stegelmann, Frank, additional, Doehner, Konstanze, additional, Lehmann, Thomas, additional, Bonatz, Karen, additional, Reiter, Andreas, additional, Boyer, Francoise, additional, Etienne, Gabriel, additional, Ianotto, Jean Christophe, additional, Ranta, Dana, additional, Roy, Lydia, additional, Cahn, Jean-Yves, additional, Harrison, Claire N., additional, Radia, Deepti, additional, Muxi, Pablo J., additional, Maldonado, Norman I, additional, Besses, Carlos, additional, Cervantes, Francisco, additional, Johansson, Peter L., additional, Barbui, Tiziano, additional, Barosi, Giovanni, additional, Vannucchi, Alessandro M., additional, Passamonti, Francesco, additional, Andreasson, Bjorn, additional, Ferrari, Maria, additional, Rambaldi, Alessandro, additional, Samuelsson, Jan, additional, Cannon, Keith, additional, Sun, Xiujuan, additional, Xu, Junqing, additional, Zhang, Peihong, additional, Xu, Zefeng, additional, and Mesa, Ruben A., additional

Published
2016
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26. Symptomatic Profiles of Patients With Polycythemia Vera : Implications of Inadequately Controlled Disease

Author

Geyer, Holly, Scherber, Robyn, Kosiorek, Heidi, Dueck, Amylou C, Kiladjian, Jean-Jacques, Xiao, Zhijian, Slot, Stefanie, Zweegman, Sonja, Sackmann, Federico, Fuentes, Ana Kerguelen, Hernández-Maraver, Dolores, Döhner, Konstanze, Harrison, Claire N, Radia, Deepti, Muxi, Pablo, Besses, Carlos, Cervantes, Francisco, Johansson, Peter L, Andreasson, Bjorn, Rambaldi, Alessandro, Barbui, Tiziano, Bonatz, Karin, Reiter, Andreas, Boyer, Francoise, Etienne, Gabriel, Ianotto, Jean-Christophe, Ranta, Dana, Roy, Lydia, Cahn, Jean-Yves, Maldonado, Norman, Barosi, Giovanni, Ferrari, Maria L, Gale, Robert Peter, Birgegard, Gunnar, Xu, Zefeng, Zhang, Yue, Sun, Xiujuan, Xu, Junqing, Zhang, Peihong, Te Boekhorst, Peter A W, Commandeur, Suzan, Schouten, Harry, Pahl, Heike L, Griesshammer, Martin, Stegelmann, Frank, Lehmann, Thomas, Senyak, Zhenya, Vannucchi, Alessandro M, Passamonti, Francesco, Samuelsson, Jan, Mesa, Ruben A, Geyer, Holly, Scherber, Robyn, Kosiorek, Heidi, Dueck, Amylou C, Kiladjian, Jean-Jacques, Xiao, Zhijian, Slot, Stefanie, Zweegman, Sonja, Sackmann, Federico, Fuentes, Ana Kerguelen, Hernández-Maraver, Dolores, Döhner, Konstanze, Harrison, Claire N, Radia, Deepti, Muxi, Pablo, Besses, Carlos, Cervantes, Francisco, Johansson, Peter L, Andreasson, Bjorn, Rambaldi, Alessandro, Barbui, Tiziano, Bonatz, Karin, Reiter, Andreas, Boyer, Francoise, Etienne, Gabriel, Ianotto, Jean-Christophe, Ranta, Dana, Roy, Lydia, Cahn, Jean-Yves, Maldonado, Norman, Barosi, Giovanni, Ferrari, Maria L, Gale, Robert Peter, Birgegard, Gunnar, Xu, Zefeng, Zhang, Yue, Sun, Xiujuan, Xu, Junqing, Zhang, Peihong, Te Boekhorst, Peter A W, Commandeur, Suzan, Schouten, Harry, Pahl, Heike L, Griesshammer, Martin, Stegelmann, Frank, Lehmann, Thomas, Senyak, Zhenya, Vannucchi, Alessandro M, Passamonti, Francesco, Samuelsson, Jan, and Mesa, Ruben A

Abstract

PURPOSE: Polycythemia vera (PV) is a myeloproliferative neoplasm (MPN) associated with 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 patients with PV who 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.

Published
2016
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27. The role of sexuality symptoms in myeloproliferative neoplasm symptom burden and quality of life : An analysis by the MPN QOL International Study Group

Author

Geyer, Holly L., Andreasson, Bjorn, Kosiorek, Heidi E., Dueck, Amylou C., Scherber, Robyn M., Martin, Kari A., Butler, Kristina A., Harrison, Claire N., Radia, Deepti H., Cervantes, Francisco, Kiladjian, Jean-Jacques, Reiter, Andreas, Birgegård, Gunnar, Passamonti, Francesco, Senyak, Zhenya, Vannucchi, Alessandro M., Paoli, Chiara, Xiao, Zhijian, Samuelsson, Jan, Mesa, Ruben A., Geyer, Holly L., Andreasson, Bjorn, Kosiorek, Heidi E., Dueck, Amylou C., Scherber, Robyn M., Martin, Kari A., Butler, Kristina A., Harrison, Claire N., Radia, Deepti H., Cervantes, Francisco, Kiladjian, Jean-Jacques, Reiter, Andreas, Birgegård, Gunnar, Passamonti, Francesco, Senyak, Zhenya, Vannucchi, Alessandro M., Paoli, Chiara, Xiao, Zhijian, Samuelsson, Jan, and Mesa, Ruben A.

Abstract

BACKGROUNDPatients with myeloproliferative neoplasms (MPNs) including polycythemia vera, essential thrombocythemia, and myelofibrosis, are faced with oppressive symptom profiles that compromise daily functioning and quality of life. Among these symptoms, sexuality-related symptoms have emerged as particularly prominent and largely unaddressed. In the current study, the authors evaluated how sexuality symptoms from MPN relate to other patient characteristics, disease features, treatments, and symptoms. METHODSA total of 1971 patients with MPN (827 with essential thrombocythemia, 682 with polycythemia vera, 456 with myelofibrosis, and 6 classified as other) were prospectively evaluated and patient responses to the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) and the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC-QLQ C30) were collected, along with information regarding individual disease characteristics and laboratory data. Sexuality scores were compared with an age-matched, healthy control population. RESULTSOverall, patients with MPN were found to have greater sexual dysfunction compared with the healthy population (MPN-SAF score of 3.6 vs 2.0; P<.001), with 64% of patients with MPN describing some degree of sexual dysfunction and 43% experiencing severe symptoms. The presence of sexual symptoms correlated closely with all domains of patient functionality (physical, social, cognitive, emotional, and role functioning) and were associated with a reduced quality of life. Sexual problems also were found to be associated with other MPN symptoms, particularly depression and nocturnal and microvascular-related symptoms. Sexual dysfunction was more severe in patients aged >65 years and in those with cytopenias and transfusion requirements, and those receiving certain therapies such as immunomodulators or steroids. ConclusionsThe results of the current study identify the topic of sexuality as a p

Published
2016
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28. Associations between gender, disease features and symptom burden in patients with myeloproliferative neoplasms: an analysis by the MPN QOL International Working Group

Author

Geyer, Holly L., primary, Kosiorek, Heidi, additional, Dueck, Amylou C., additional, Scherber, Robyn, additional, Slot, Stefanie, additional, Zweegman, Sonja, additional, te Boekhorst, Peter AW, additional, Senyak, Zhenya, additional, Schouten, Harry C., additional, Sackmann, Federico, additional, Fuentes, Ana Kerguelen, additional, Hernández-Maraver, Dolores, additional, Pahl, Heike L., additional, Griesshammer, Martin, additional, Stegelmann, Frank, additional, Döhner, Konstanze, additional, Lehmann, Thomas, additional, Bonatz, Karin, additional, Reiter, Andreas, additional, Boyer, Francoise, additional, Etienne, Gabriel, additional, Ianotto, Jean-Christophe, additional, Ranta, Dana, additional, Roy, Lydia, additional, Cahn, Jean-Yves, additional, Harrison, Claire N., additional, Radia, Deepti, additional, Muxi, Pablo, additional, Maldonado, Norman, additional, Besses, Carlos, additional, Cervantes, Francisco, additional, Johansson, Peter L., additional, Barbui, Tiziano, additional, Barosi, Giovanni, additional, Vannucchi, Alessandro M., additional, Paoli, Chiara, additional, Passamonti, Francesco, additional, Andreasson, Bjorn, additional, Ferrari, Maria L, additional, Rambaldi, Alessandro, additional, Samuelsson, Jan, additional, Cannon, Keith, additional, Birgegard, Gunnar, additional, Xiao, Zhijian, additional, Xu, Zefeng, additional, Zhang, Yue, additional, Sun, Xiujuan, additional, Xu, Junqing, additional, Kiladjian, Jean-Jacques, additional, Zhang, Peihong, additional, Gale, Robert Peter, additional, and Mesa, Ruben A., additional

Published
2016
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29. The role of sexuality symptoms in myeloproliferative neoplasm symptom burden and quality of life: An analysis by the MPN QOL International Study Group

Author

Geyer, Holly L., primary, Andreasson, Bjorn, additional, Kosiorek, Heidi E., additional, Dueck, Amylou C., additional, Scherber, Robyn M., additional, Martin, Kari A., additional, Butler, Kristina A., additional, Harrison, Claire N., additional, Radia, Deepti H., additional, Cervantes, Francisco, additional, Kiladjian, Jean‐Jacques, additional, Reiter, Andreas, additional, Birgegard, Gunnar, additional, Passamonti, Francesco, additional, Senyak, Zhenya, additional, Vannucchi, Alessandro M., additional, Paoli, Chiara, additional, Xiao, Zhijian, additional, Samuelsson, Jan, additional, and Mesa, Ruben A., additional

Published
2016
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30. Symptomatic Profiles of Patients With Polycythemia Vera: Implications of Inadequately Controlled Disease

Author

Geyer, Holly, primary, Scherber, Robyn, additional, Kosiorek, Heidi, additional, Dueck, Amylou C., additional, Kiladjian, Jean-Jacques, additional, Xiao, Zhijian, additional, Slot, Stefanie, additional, Zweegman, Sonja, additional, Sackmann, Federico, additional, Fuentes, Ana Kerguelen, additional, Hernández-Maraver, Dolores, additional, Döhner, Konstanze, additional, Harrison, Claire N., additional, Radia, Deepti, additional, Muxi, Pablo, additional, Besses, Carlos, additional, Cervantes, Francisco, additional, Johansson, Peter L., additional, Andreasson, Bjorn, additional, Rambaldi, Alessandro, additional, Barbui, Tiziano, additional, Bonatz, Karin, additional, Reiter, Andreas, additional, Boyer, Francoise, additional, Etienne, Gabriel, additional, Ianotto, Jean-Christophe, additional, Ranta, Dana, additional, Roy, Lydia, additional, Cahn, Jean-Yves, additional, Maldonado, Norman, additional, Barosi, Giovanni, additional, Ferrari, Maria L., additional, Gale, Robert Peter, additional, Birgegard, Gunnar, additional, Xu, Zefeng, additional, Zhang, Yue, additional, Sun, Xiujuan, additional, Xu, Junqing, additional, Zhang, Peihong, additional, te Boekhorst, Peter A.W., additional, Commandeur, Suzan, additional, Schouten, Harry, additional, Pahl, Heike L., additional, Griesshammer, Martin, additional, Stegelmann, Frank, additional, Lehmann, Thomas, additional, Senyak, Zhenya, additional, Vannucchi, Alessandro M., additional, Passamonti, Francesco, additional, Samuelsson, Jan, additional, and Mesa, Ruben A., additional

Published
2016
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31. Unmet Needs for Symptom Control in Essential Thrombocythemia with Front Line Therapy

Author

Geyer, Holly, primary, Scherber, Robyn M., additional, Kosiorek, Heidi, additional, Dueck, Amylou C., additional, Kiladjian, Jean-Jacques, additional, Slot, Stefanie, additional, Xiao, Zhijian, additional, Zweegman, Sonja, additional, Sackmann, Federico, additional, Kerguelen Fuentes, Ana, additional, Hernandez-Maraver, Dolores, additional, Döhner, Konstanze, additional, Harrison, Claire N., additional, Radia, Deepti, additional, Muxi, Pablo J., additional, Besses, Carlos, additional, Cervantes, Francisco, additional, Johansson, Peter L., additional, Andreasson, Bjorn, additional, Rambaldi, Alessandro, additional, Barbui, Tiziano, additional, Bonatz, Karin, additional, Reiter, Andreas, additional, Boyer, Francoise, additional, Etienne, Gabriel, additional, Ianotto, Jean-Christophe, additional, Ranta, Dana, additional, Roy, Lydia, additional, Cahn, Jean-Yves, additional, Maldonado, Norman I, additional, Barosi, Giovanni, additional, Ferrari, Maria, additional, Gale, Robert Peter, additional, Birgegard, Gunnar, additional, Xu, Zefeng, additional, Zhang, Yu, additional, Sun, Xiujuan, additional, Xu, Junqing, additional, Zhang, Peihong, additional, te Boekhorst, Peter A., additional, Commandeur, Suzan, additional, Schouten, Harry C., additional, Pahl, Heike L, additional, Griesshammer, Martin, additional, Stegelmann, Frank, additional, Lehmann, Thomas, additional, Vannucchi, Alessandro M., additional, Passamonti, Francesco, additional, Samuelsson, Jan, additional, and Mesa, Ruben A., additional

Published
2015
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33. Impact of Disease Duration upon Symptom Burden Amongst Patients with Myeloproliferative Neoplasms (MPNs)

Author

Scherber, Robyn M., primary, Geyer, Holly, additional, Harrison, Claire N., additional, Kosiorek, Heidi E., additional, Dueck, Amylou C., additional, Kiladjian, Jean-Jacques, additional, Slot, Stefanie, additional, Xiao, Zhijian, additional, Zweegman, Sonja, additional, Sackmann, Federico, additional, Kerguelen Fuentes, Ana, additional, Hernandez-Maraver, Dolores, additional, Döhner, Konstanze, additional, Radia, Deepti, additional, Muxi, Pablo J., additional, Besses, Carlos, additional, Cervantes, Francisco, additional, Johansson, Peter L., additional, Andreasson, Bjorn, additional, Rambaldi, Alessandro, additional, Barbui, Tiziano, additional, Bonatz, Karin, additional, Reiter, Andreas, additional, Boyer, Francoise, additional, Etienne, Gabriel, additional, Ianotto, Jean-Christophe, additional, Ranta, Dana, additional, Roy, Jean, additional, Cahn, Jean-Yves, additional, Maldonado, Norman I, additional, Barosi, Giovanni, additional, Ferrari, Maria, additional, Gale, Robert Peter, additional, Birgegard, Gunnar, additional, Xu, Zefeng, additional, Zhang, Yu, additional, Sun, Xiujuan, additional, Xu, Junqing, additional, Zhang, Peihong, additional, te Boekhorst, Peter A., additional, Commandeur, Suzan, additional, Schouten, Harry C., additional, Pahl, Heike L, additional, Griesshammer, Martin, additional, Stegelmann, Frank, additional, Lehmann, Thomas, additional, Vannucchi, Alessandro M., additional, Passamonti, Francesco, additional, Samuelsson, Jan, additional, and Mesa, Ruben A., additional

Published
2015
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34. Symptom Burden Profile in Myelofibrosis Patients with Thrombocytopenia: Lessons and Unmet Needs

Author

Geyer, Holly, primary, Scherber, Robyn M, additional, Kosiorek, Heidi, additional, Dueck, Amylou C., additional, Kiladjian, Jean-Jacques, additional, Xiao, Zhijian, additional, Slot, Stefanie, additional, Zweegman, Sonja, additional, Sackmann, Federico, additional, Kerguelen Fuentes, Ana, additional, Hernandez-Maraver, Dolores, additional, Dohner, Konstanze, additional, Harrison, Claire N., additional, Radia, Deepti, additional, Muxi, Pablo J., additional, Besses, Carlos, additional, Cervantes, Francisco, additional, Johansson, Peter L., additional, Andreasson, Bjorn, additional, Rambaldi, Alessandro, additional, Barbui, Tiziano, additional, Bonatz, Karin, additional, Reiter, Andreas, additional, Boyer, Francoise, additional, Etienne, Gabriel, additional, Ianotto, Jean-Christophe, additional, Ranta, Dana, additional, Roy, Lydia, additional, Cahn, Jean-Yves, additional, Maldonado, Norman I, additional, Barosi, Giovanni, additional, Ferrari, Maria, additional, Gale, Robert Peter, additional, Birgegard, Gunnar, additional, Xu, Zefeng, additional, Zhang, Yue, additional, Sun, Xiujuan, additional, Xu, Junqing, additional, Zhang, Peihong, additional, te Boekhorst, Peter, additional, Commandeur, Suzan, additional, Schouten, Harry C., additional, Pahl, Heike L, additional, Griesshammer, Martin, additional, Stegelmann, Frank, additional, Lehmann, Thomas, additional, Vannucchi, Alessandro M., additional, Passamonti, Francesco, additional, Samuelsson, Jan, additional, and Mesa, Ruben A., additional

Published
2015
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35. A Study of the Role of Antiplatelet Therapy in the Prevention of Thrombosis in Patients with Calr-Mutated Low Risk Essential Thrombocythemia

Author

Alvarez-Larrán, Alberto, primary, Guglielmelli, Paola, additional, Arellano-Rodrigo, Eduardo, additional, Griesshammer, Martin, additional, Paoli, Chiara, additional, Kerguelen, Ana, additional, Ferrer-Marin, Francisca, additional, Hernández-Boluda, Juan Carlos, additional, Andreasson, Bjorn, additional, Schwarz, Jiri, additional, García-Gutierrez, Valentín, additional, Ayala, Rosa M., additional, Barba, Pere, additional, Gómez-Casares, María Teresa, additional, Skoda, Radek C., additional, Burgaleta, Carmen, additional, Slot, Stefanie, additional, Samuelsson, Jan, additional, Samah, Alimam, additional, Beauverd, Yan, additional, Harrison, Claire N., additional, Cervantes, Francisco, additional, Vannucchi, Alessandro M., additional, and Besses, Carlos, additional

Published
2015
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36. Myeloproliferative Neoplasm Quality of Life (MPN-QOL) Study Group: Results from the MPN Experimental Assessment of Symptoms By Utilizing Repetitive Evaluation (MEASURE) Trial

Author

Scotch, Allison, Scherber, Robyn M., Bruso, Marlene, Kosiorek, Heidi Elizabeth, Dueck, Amylou C., Geyer, Holly, Fauble, Veena D.S., Vannucchi, Alessandro M., Fuentes, Ana Kerguelen, Andersen, Christen Lykkegaard, Ejerblad, Elisabeth, Birgegard, Gunnar, Johansson, Peter L., Andreasson, Bjorn, Ross, David M, Sackman, Federico, Demory, Jean-Loup, Tam, Constantine S., Michiels, Jan J., Radia, Deepti, Ewing, Joanne, Wadelin, Frances, Lown, Robert N., McMullin, Mary Frances, Swe, Wunna, Cullis, Jonathan O., Sargant, Nigel, Moreton, Paul, Cargo, Catherine, Sekhar, Mallika, Shekouhi, Satareh, and Mesa, Ruben A

Published
2017
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37. Distinct clustering of symptomatic burden among myeloproliferative neoplasm patients : retrospective assessment in 1470 patients

Author

Geyer, Holly L., Scherber, Robyn M., Dueck, Amylou C., Kiladjian, Jean-Jacques, Xiao, Zhijian, Slot, Stefanie, Zweegman, Sonja, Sackmann, Federico, Kerguelen Fuentes, Ana, Hernandez-Maraver, Dolores, Doehner, Konstanze, Harrison, Claire N., Radia, Deepti, Muxi, Pablo, Besses, Carlos, Cervantes, Francisco, Johansson, Peter L., Andreasson, Bjorn, Rambaldi, Alessandro, Barbui, Tiziano, Vannucchi, Alessandro M., Passamonti, Francesco, Samuelsson, Jan, Birgegård, Gunnar, Mesa, Ruben A., Geyer, Holly L., Scherber, Robyn M., Dueck, Amylou C., Kiladjian, Jean-Jacques, Xiao, Zhijian, Slot, Stefanie, Zweegman, Sonja, Sackmann, Federico, Kerguelen Fuentes, Ana, Hernandez-Maraver, Dolores, Doehner, Konstanze, Harrison, Claire N., Radia, Deepti, Muxi, Pablo, Besses, Carlos, Cervantes, Francisco, Johansson, Peter L., Andreasson, Bjorn, Rambaldi, Alessandro, Barbui, Tiziano, Vannucchi, Alessandro M., Passamonti, Francesco, Samuelsson, Jan, Birgegård, Gunnar, and Mesa, Ruben A.

Abstract

Symptom burden in myeloproliferative neoplasms (MPNs) is heterogeneous even among patients within the same MPN diagnosis. Using cluster analysis from prospectively gathered symptom burden data in 1470 international patients with essential thrombocythemia (ET), polycythemia vera (PV), or myelofibrosis (MF), we assessed for the presence of clusters and relationship to disease features and prognosis. In MF (4 clusters identified), clusters significantly differed by Dynamic International Prognostic Scoring System (DIPSS) risk (P < .001), leukopenia (P = .009), thrombocytopenia (P < .001), and spleen size (P = .02). Although an association existed between clusters and DIPSS risk, high symptom burden was noted in some low and intermediate-1-risk MF patients. In PV (5 clusters identified), total symptom score increased across clusters (P < .001), but clusters did not significantly differ by PV risk or the risk assessment variable of age. Among ET patients (5 clusters identified), clusters differed by gender (P = .04), anemia (P = .01), and prior hemorrhage (P = .047). Total symptom score increased across clusters (P < .001), but clusters did not significantly differ by International Prognostic Score for ET risk including the risk assessment variables. Significant symptom heterogeneity exists within each MPN subtype, sometimes independent of disease features or prognosis.

Published
2014
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38. A Retrospective Cohort Study of Interferon-alpha Therapy in Myeloproliferative Neoplasms; Adverse Events, Thromboembolic Incidence and Causes of Termination of Therapy

Author

Lindgren, Marie, Samuelsson, Jan, Nilsson, Lars, Knutsen, Havar, Ghanima, Waleed, Astrom, Maria, Ejerblad, Elisabeth, Hugosson, Anna, Johansson, Peter L., Andreasson, Bjorn, Lindgren, Marie, Samuelsson, Jan, Nilsson, Lars, Knutsen, Havar, Ghanima, Waleed, Astrom, Maria, Ejerblad, Elisabeth, Hugosson, Anna, Johansson, Peter L., and Andreasson, Bjorn

Published
2014

39. Symptom Severity and Clinical Variables of Polycythemia Vera Patients with Splenomegaly, Phlebotomy Requirements and/or Hydroxyurea Use : a Retrospective Evaluation of 1334 Patients

Author

Geyer, Holly Lynn, Scherber, Robyn M., Dueck, Amylou Constance, Kiladjian, Jean-Jacques, Xiao, Zhijian, Slot, Stephanie, Zweegman, Sonja, Kerguelen Fuentes, Ana, Hernandez-Maraver, Dolores, Dohner, Konstanze, Harrison, Claire N., Radia, Deepti H., Muxi, Pablo J., Sackman, Federico, Besses, Carlos, Cervantes, Francisco, Johansson, Peter L., Andreasson, Bjorn, Rambaldi, Alessandro, Barbui, Tiziano, Vannucchi, Alessandro M., Passamonti, Francesco, Samuelsson, Jan, Birgegård, Gunnar, Bonatz, Karin, Reiter, Andreas, Boyer, Francoise, Etienne, Gabriel, Ianotto, Jean-Christophe, Ranta, Dana, Roy, Lydia, Cahn, Jean-Yves, Maldonado, Norman I., Barosi, Giovanni, Ferrari, Maria, Cannon, Keith, te Boekhorst, Peter, Schouten, Harry C., Pahl, Heike L., Griesshammer, Martin, Stegelmann, Frank, Lehmann, Thomas, Xu, Zefeng, Zhang, Yue, Sun, Xiujuan, Xu, Junqing, Zhang, Peihong, Mesa, Ruben, Geyer, Holly Lynn, Scherber, Robyn M., Dueck, Amylou Constance, Kiladjian, Jean-Jacques, Xiao, Zhijian, Slot, Stephanie, Zweegman, Sonja, Kerguelen Fuentes, Ana, Hernandez-Maraver, Dolores, Dohner, Konstanze, Harrison, Claire N., Radia, Deepti H., Muxi, Pablo J., Sackman, Federico, Besses, Carlos, Cervantes, Francisco, Johansson, Peter L., Andreasson, Bjorn, Rambaldi, Alessandro, Barbui, Tiziano, Vannucchi, Alessandro M., Passamonti, Francesco, Samuelsson, Jan, Birgegård, Gunnar, Bonatz, Karin, Reiter, Andreas, Boyer, Francoise, Etienne, Gabriel, Ianotto, Jean-Christophe, Ranta, Dana, Roy, Lydia, Cahn, Jean-Yves, Maldonado, Norman I., Barosi, Giovanni, Ferrari, Maria, Cannon, Keith, te Boekhorst, Peter, Schouten, Harry C., Pahl, Heike L., Griesshammer, Martin, Stegelmann, Frank, Lehmann, Thomas, Xu, Zefeng, Zhang, Yue, Sun, Xiujuan, Xu, Junqing, Zhang, Peihong, and Mesa, Ruben

Published
2014

40. Symptom Severity and Clinical Variables of Polycythemia Vera Patients with Splenomegaly, Phlebotomy Requirements and/or Hydroxyurea Use: a Retrospective Evaluation of 1334 Patients

Author

Geyer, Holly Lynn, primary, Scherber, Robyn M, additional, Dueck, Amylou Constance, additional, Kiladjian, Jean-Jacques, additional, Xiao, Zhijian, additional, Slot, Stephanie, additional, Zweegman, Sonja, additional, Fuentes, Ana Kerguelen, additional, Hernández-Maraver, Dolores, additional, Dohner, Konstanze, additional, Harrison, Claire N., additional, Radia, Deepti H, additional, Muxi, Pablo J., additional, Sackman, Federico, additional, Besses, Carlos, additional, Cervantes, Francisco, additional, Johansson, Peter L., additional, Andreasson, Bjorn, additional, Rambaldi, Alessandro, additional, Barbui, Tiziano, additional, Vannucchi, Alessandro M., additional, Passamonti, Francesco, additional, Samuelsson, Jan, additional, Birgegard, Gunnar, additional, Bonatz, Karin, additional, Reiter, Andreas, additional, Boyer, Françoise, additional, Etienne, Gabriel, additional, Ianotto, Jean-Christophe, additional, Ranta, Dana, additional, Roy, Lydia, additional, Cahn, Jean-Yves, additional, Maldonado, Norman I, additional, Barosi, Giovanni, additional, Ferrari, Maria, additional, Cannon, Keith, additional, te Boekhorst, Peter, additional, Schouten, Harry C, additional, Pahl, Heike L., additional, Griesshammer, Martin, additional, Stegelmann, Frank, additional, Lehmann, Thomas, additional, Xu, Zefeng, additional, Zhang, Yue, additional, Sun, Xiujuan, additional, Xu, Junqing, additional, Zhang, Peihong, additional, and Mesa, Ruben, additional

Published
2014
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41. Towards a Better Understanding of Epidemiology, Survival and Treatment in Myeloproliferative Neoplasms: Results of the European Leukemianet Registry (ERNEST study)

Author

Barbui, Tiziano, primary, Masciulli, Arianna, additional, Scarano, Marco, additional, Tognoni, Gianni, additional, Sisti, Sara, additional, Di Lelio, Alessandra, additional, Vannucchi, Alessandro M., additional, Cazzola, Mario, additional, Rambaldi, Alessandro, additional, Doehener, Konstanze, additional, Cervantes, Francisco, additional, Passamonti, Francesco, additional, Reiter, Andreas, additional, Koschmieder, Steffen, additional, Niederwieser, Dietger, additional, Andreasson, Bjorn, additional, Johansson, Peter L., additional, Hochhaus, Andreas, additional, and Harrison, Claire N., additional

Published
2014
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42. MPD-RC 101 prospective study of reduced-intensity allogeneic hematopoietic stem cell transplantation in patients with myelofibrosis

Author

Rondelli, Damiano, primary, Goldberg, Judith D., additional, Isola, Luis, additional, Price, Leah S., additional, Shore, Tsiporah B., additional, Boyer, Michael, additional, Bacigalupo, Andrea, additional, Rambaldi, Alessandro, additional, Scarano, Marco, additional, Klisovic, Rebecca B., additional, Gupta, Vikas, additional, Andreasson, Bjorn, additional, Mascarenhas, John, additional, Wetzler, Meir, additional, Vannucchi, Alessandro M., additional, Prchal, Josef T., additional, Najfeld, Vesna, additional, Orazi, Attilio, additional, Weinberg, Rona S., additional, Miller, Crystal, additional, Barosi, Giovanni, additional, Silverman, Lewis R., additional, Prosperini, Giuseppe, additional, Marchioli, Roberto, additional, and Hoffman, Ronald, additional

Published
2014
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43. Distinct clustering of symptomatic burden among myeloproliferative neoplasm patients: retrospective assessment in 1470 patients

Author

Geyer, Holly L., primary, Scherber, Robyn M., additional, Dueck, Amylou C., additional, Kiladjian, Jean-Jacques, additional, Xiao, Zhijian, additional, Slot, Stefanie, additional, Zweegman, Sonja, additional, Sackmann, Federico, additional, Fuentes, Ana Kerguelen, additional, Hernández-Maraver, Dolores, additional, Döhner, Konstanze, additional, Harrison, Claire N., additional, Radia, Deepti, additional, Muxi, Pablo, additional, Besses, Carlos, additional, Cervantes, Francisco, additional, Johansson, Peter L., additional, Andreasson, Bjorn, additional, Rambaldi, Alessandro, additional, Barbui, Tiziano, additional, Vannucchi, Alessandro M., additional, Passamonti, Francesco, additional, Samuelsson, Jan, additional, Birgegard, Gunnar, additional, and Mesa, Ruben A., additional

Published
2014
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44. Patients with polycythemia vera have worst impairment of quality of life among patients with newly diagnosed myeloproliferative neoplasms

Author

Abelsson, Johanna, Andreasson, Bjorn, Samuelsson, Jan, Hultcrantz, Malin, Ejerblad, Elisabeth, Johansson, Berit, Emanuel, Robyn, Mesa, Ruben, Johansson, Peter, Abelsson, Johanna, Andreasson, Bjorn, Samuelsson, Jan, Hultcrantz, Malin, Ejerblad, Elisabeth, Johansson, Berit, Emanuel, Robyn, Mesa, Ruben, and Johansson, Peter

Abstract

The quality of life (QoL) at the time of diagnosis of myeloproliferative neoplasm (MPN) has, to date, not been studied. One hundred and seventy-nine patients with MPN: 80 with essential thrombocythemia (ET), 73 with polycythemia vera (PV), 22 with primary myelofibrosis (PMF) and four with MPN undifferentiated, were included in this study. European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC-QLQC30) and the MPN-Symptom Assessment Form (MPN-SAF) were used to evaluate QoL. Fatigue was the most reported symptom in these patients. Patients with PV reported significantly higher mean scores for inactivity, dizziness, cough, itching, depression and lower total QoL compared to patients with ET. Patients with PV had significantly more headache and itching compared to patients with PMF. When the newly diagnosed patients with MPN were compared with a cohort of patients with MPN with mean disease duration of 7.8 years, the differences were most striking for patients with PMF, with significantly more fatigue, abdominal discomfort, concentration problems, insomnia, fever, weight loss and lower overall QoL developed over time.

Published
2013
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45. A phase II study of vorinostat (MK-0683) in patients with polycythaemia vera and essential thrombocythaemia

Author

Andersen, Christen L., McMullin, Mary F., Ejerblad, Elisabeth, Zweegman, Sonja, Harrison, Claire, Fernandes, Savio, Bareford, David, Knapper, Steven, Samuelsson, Jan, Loefvenberg, Eva, Linder, Olle, Andreasson, Bjorn, Ahlstrand, Erik, Jensen, Morten K., Bjerrum, Ole W., Vestergaard, Hanne, Larsen, Herdis, Klausen, Tobias W., Mourits-Andersen, Torben, Hasselbalch, Hans C., Andersen, Christen L., McMullin, Mary F., Ejerblad, Elisabeth, Zweegman, Sonja, Harrison, Claire, Fernandes, Savio, Bareford, David, Knapper, Steven, Samuelsson, Jan, Loefvenberg, Eva, Linder, Olle, Andreasson, Bjorn, Ahlstrand, Erik, Jensen, Morten K., Bjerrum, Ole W., Vestergaard, Hanne, Larsen, Herdis, Klausen, Tobias W., Mourits-Andersen, Torben, and Hasselbalch, Hans C.

Abstract

Inhibition of histone deacetylases may be an important target in patients with myeloproliferative neoplasms. This investigator-initiated, non-randomized, open-label phase II multi-centre study included 63 patients (19 essential thrombocythaemia, 44 polycythaemia vera) from 15 centres. The primary objective was to evaluate if vorinostat was followed by a decline in clonal myeloproliferation as defined by European Leukaemia Net. Thirty patients (48%) completed the intervention period (24 weeks of therapy). An intention-to-treat response rate of 35% was identified. Pruritus was resolved [19% to 0% (P=0.06)] and the prevalence of splenomegaly was lowered from 50% to 27% (P=0.03). Sixty-five per cent of the patients experienced a decrease in JAK2 V617F allele burden (P=0.006). Thirty-three patients (52% of patients) discontinued study drug before end of intervention due to adverse events (28 patients) or lack of response (5 patients). In conclusion, vorinostat showed effectiveness by normalizing elevated leucocyte and platelet counts, resolving pruritus and significantly reducing splenomegaly. However, vorinostat was associated with significant side effects resulting in a high discontinuation rate. A lower dose of vorinostat in combination with conventional and/or novel targeted therapies may be warranted in future studies.

Published
2013
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46. Myeloproliferative Neoplasm Quality Of Life (MPN-QOL) Study Group: Observational Study Of Quality Of Life and Symptomatic Response In Myelofibrosis Patients Receiving Undergoing Treatment With Conventional Therapy, The Measures Trial and Allogeneic Stem Cell Transplant, The Symptoms Trial

Author

Fauble, Veena, primary, Emanuel, Robyn M., additional, Geyer, Holly Lynn, additional, Dueck, Amylou Constance, additional, Kroeger, Nicolaus, additional, Scott, Bart L, additional, Gerds, Aaron T, additional, Popat, Uday R, additional, Giralt, Sergio A, additional, Gupta, Vikas, additional, Ross, David, additional, Knight, Emily, additional, Fuentes, Ana Kerguelen, additional, Andersen, Christen Lykkegaard, additional, Ejerblad, Elisabeth, additional, Birgegard, Gunnar, additional, Johansson, Peter, additional, Andreasson, Bjorn, additional, Sackmann, Federico, additional, Demory, Jean-Loup, additional, Tam, Constantine S, additional, Michiels, Jan J., additional, Radia, Deepti H, additional, Boxer, Michael, additional, and Mesa, Ruben A., additional

Published
2013
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47. Insomnia, Quality Of Life and MPN Symptom Burden: An Analysis By The MPN Quality Of Life International Study Group (MPN-QOL ISG)

Author

Geyer, Holly Lynn, primary, Dueck, Amylou Constance, additional, Emanuel, Robyn M., additional, Cannon, Keith, additional, Kiladjian, Jean-Jacques, additional, Slot, Stephanie, additional, Zweegman, Sonja, additional, Boekhorst, Peter, additional, Commandeur, Suzan, additional, Schouten, Harry C., additional, Sackmann, Federico, additional, Fuentes, Ana Kerguelen, additional, Hernández-Maraver, Dolores, additional, Pahl, Heike L., additional, Griesshammer, Martin, additional, Stegelmann, Frank, additional, Dohner, Konstanze, additional, Lehmann, Thomas, additional, Bonatz, Karin, additional, Reiter, Andreas, additional, Boyer, Françoise, additional, Etienne, Gabriel, additional, Ianotto, Jean-Christophe, additional, Ranta, Dana, additional, Roy, Lydia, additional, Cahn, Jean-Yves, additional, Harrison, Claire N, additional, Radia, Deepti H, additional, Muxi, Pablo J., additional, Maldonado, Norman I, additional, Besses, Carlos, additional, Cervantes, Francisco, additional, Johansson, Peter, additional, Barbui, Tiziano, additional, Barosi, Giovanni, additional, Vannucchi, Alessandro M., additional, Passamonti, Francesco, additional, Andreasson, Bjorn, additional, Ferrari, Maria, additional, Rambaldi, Alessandro, additional, Samuelsson, Jan, additional, Birgegard, Gunnar, additional, Xiao, Zhijian, additional, Xu, Zefeng, additional, Zhang, Yue, additional, Sun, Xiujuan, additional, Xu, Junqing, additional, Zhang, Peihong, additional, Peter Gale, Robert Peter, additional, and Mesa, Ruben A., additional

Published
2013
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48. Sexuality Challenges, Intimacy, and MPN Symptom Burden: An Analysis By The MPN Quality Of Life International Study Group (MPN-QOL ISG)

Author

Geyer, Holly Lynn, primary, Dueck, Amylou C., additional, Emanuel, Robyn M., additional, Cannon, Keith, additional, Kiladjian, Jean-Jacques, additional, Slot, Stephanie, additional, Zweegman, Sonja, additional, Boekhorst, Peter, additional, Commandeur, Suzan, additional, Schouten, Harry C., additional, Sackmann, Federico, additional, Fuentes, Ana Kerguelen, additional, Hernandez, Dolores, additional, Pahl, Heike L., additional, Griesshammer, Martin, additional, Stegelmann, Frank, additional, Döhner, Konstanze, additional, Lehmann, Thomas, additional, Ranta, Dana, additional, Reiter, Andreas, additional, Boyer, Françoise, additional, Etienne, Gabriel, additional, Ianotto, Jean-Christophe, additional, Roy, Lydia, additional, Cahn, Jean-Yves, additional, Harrison, Claire N, additional, Radia, Deepti H, additional, Muxi, Pablo J., additional, Maldonado, Norman I, additional, Besses, Carlos, additional, Cervantes, Francisco, additional, Johansson, Peter, additional, Barbui, Tiziano, additional, Barosi, Giovanni, additional, Vannucchi, Alessandro M, additional, Passamonti, Francesco, additional, Andreasson, Bjorn, additional, Ferrari, Maria, additional, Rambaldi, Alessandro, additional, Samuelsson, Jan, additional, Birgegard, Gunnar, additional, Xiao, Zhijian, additional, Xu, Zefeng, additional, Sun, Xiujuan, additional, Xu, Junqing, additional, Zhang, Peihong, additional, Gale, Robert Peter, additional, and Mesa, Ruben A, additional

Published
2013
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49. Myeloproliferative (MPN) Symptom Burden Response Thresholds: Assessment Of MPN-SAF TSS Quartiles As Potential Markers Of Symptom Response

Author

Emanuel, Robyn M., primary, Dueck, Amylou Constance, additional, Geyer, Holly Lynn, additional, Kiladjian, Jean-Jacques, additional, Slot, Stephanie, additional, Zweegman, Sonja, additional, Boekhorst, Peter te, additional, Commandeur, Suzan, additional, Schouten, Harry C., additional, Sackmann, Federico, additional, Hernandez, Dolores, additional, Fuentes, Ana Kerguelen, additional, Pahl, Heike L., additional, Stegelmann, Frank, additional, Doehner, Konstanze, additional, Lehmann, Thomas, additional, Bonatz, Karin, additional, Reiter, Andreas, additional, Boyer, Françoise, additional, Etienne, Gabriel, additional, Ianotto, Jean-Christophe, additional, Ranta, Dana, additional, Roy, Lydia, additional, Cahn, Jean-Yves, additional, Harrison, Claire N, additional, Radia, Deepti H, additional, Muxi, Pablo J., additional, Maldonado, Norman I, additional, Besses, Carlos, additional, Cervantes, Francisco, additional, Johansson, Peter, additional, Barbui, Tiziano, additional, Barosi, Giovanni, additional, Vannucchi, Alessandro M, additional, Passamonti, Francesco, additional, Andreasson, Bjorn, additional, Ferrari, Maria, additional, Griesshammer, Martin, additional, Rambaldi, Alessandro, additional, Samuelsson, Jan, additional, Birgegard, Gunnar, additional, Xiao, Zhijian, additional, Xu, Zefeng, additional, Zhang, Yue, additional, Sun, Xiujuan, additional, Xu, Junqing, additional, Zhang, Peihong, additional, Gale, Robert Peter, additional, Tefferi, Ayalew, additional, and Mesa, Ruben A., additional

Published
2013
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50. Thalidomide and dexamethasone vs. bortezomib and dexamethasone for melphalan refractory myeloma : a randomized study

Author

Hjorth, Martin, Hjertner, Oyvind, Knudsen, Lene Meldgaard, Gulbrandsen, Nina, Holmberg, Erik, Pedersen, Per Trollund, Andersen, Niels Frost, Andreasson, Bjorn, Billstrom, Rolf, Carlson, Kristina, Carlsson, Margaretha S., Flogegard, Max, Forsberg, Karin, Gimsing, Peter, Karlsson, Torbjorn, Linder, Olle, Nahi, Hareth, Othzen, Annika, Swedin, Agneta, Hjorth, Martin, Hjertner, Oyvind, Knudsen, Lene Meldgaard, Gulbrandsen, Nina, Holmberg, Erik, Pedersen, Per Trollund, Andersen, Niels Frost, Andreasson, Bjorn, Billstrom, Rolf, Carlson, Kristina, Carlsson, Margaretha S., Flogegard, Max, Forsberg, Karin, Gimsing, Peter, Karlsson, Torbjorn, Linder, Olle, Nahi, Hareth, Othzen, Annika, and Swedin, Agneta

Abstract

Objectives: Thalidomide and bortezomib have been frequently used for second-line therapy in patients with myeloma relapsing after or refractory to initial melphalan-based treatment, but no randomized trials have been published comparing these two treatment alternatives. Methods: Thalidomide- and bortezomib-naive patients with melphalan refractory myeloma were randomly assigned to low-dose thalidomide + dexamethasone (Thal-Dex) or bortezomib + dexamethasone (Bort-Dex). At progression on either therapy, the patients were offered crossover to the alternative drug combination. An estimated 300 patients would be needed for the trial to detect a 50% difference in median PFS between the treatment arms. Results: After inclusion of 131 patients, the trial was prematurely closed because of low accrual. Sixty-seven patients were randomized to Thal-Dex and 64 to Bort-Dex. Progression-free survival was similar (median, 9.0 months for Thal-Dex and 7.2 for Bort-Dex). Response rate was similar (55% for Thal-Dex and 63% for Bort-Dex), but time to response was shorter (P < 0.05) and the VGPR rate higher (P < 0.01) for Bort-Dex. Time-to-other treatment after crossover was similar (median, 13.2 months for Thal-Dex and 11.2 months for Bort-Dex), as was overall survival (22.8 months for Thal-Dex and 19.0 for Bort-Dex). Venous thromboembolism was seen in seven patients and cerebrovascular events in four patients in the Thal-Dex group. Severe neuropathy, reactivation of herpes virus infections, and mental depression were more frequently observed in the Bort-Dex group. In the quality-of-life analysis, no difference was noted for physical function, pain, and global quality of life. Fatigue and sleep disturbances were significantly more prevalent in the Bort-Dex group. Conclusions: Thalidomide (50-100 mg daily) in combination with dexamethasone seems to have an efficacy comparable with that of bortezomib and dexamethasone in melphalan refractory myeloma. However, the statistical strengt

Published
2012
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