Comparison of Dynamic International Prognostic Scoring System and MYelofibrosis SECondary to PV and ET Prognostic Model for Prediction of Outcome in Polycythemia Vera and Essential Thrombocythemia Myelofibrosis after Allogeneic Stem Cell Transplantation
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Comparison of Dynamic International Prognostic Scoring System and MYelofibrosis SECondary to PV and ET Prognostic Model for Prediction of Outcome in Polycythemia Vera and Essential Thrombocythemia Myelofibrosis after Allogeneic Stem Cell Transplantation. / Gagelmann, Nico; Eikema, Diderik-Jan; de Wreede, Liesbeth C; Koster, Linda; Wolschke, Christine; Arnold, Renate; Kanz, Lothar; McQuaker, Grant; Marchand, Tony; Socié, Gerard; Bourhis, Jean Henri; Mohty, Mohamad; Cornelissen, Jan J; Chevallier, Patrice; Bernasconi, Paolo; Stelljes, Matthias; Rohrlich, Pierre-Simon; Fanin, Renato; Finke, Jürgen; Maertens, Johan; Blaise, Didier; Itälä-Remes, Maija; Labussière-Wallet, Hélène; Robin, Marie; McLornan, Donal; Chalandon, Yves; Yakoub-Agha, Ibrahim; Kröger, Nicolaus; CMWP of the European Society for Blood and Marrow Transplantation.
in: BIOL BLOOD MARROW TR, Jahrgang 25, Nr. 6, 06.2019, S. e204-e208.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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T1 - Comparison of Dynamic International Prognostic Scoring System and MYelofibrosis SECondary to PV and ET Prognostic Model for Prediction of Outcome in Polycythemia Vera and Essential Thrombocythemia Myelofibrosis after Allogeneic Stem Cell Transplantation
AU - Gagelmann, Nico
AU - Eikema, Diderik-Jan
AU - de Wreede, Liesbeth C
AU - Koster, Linda
AU - Wolschke, Christine
AU - Arnold, Renate
AU - Kanz, Lothar
AU - McQuaker, Grant
AU - Marchand, Tony
AU - Socié, Gerard
AU - Bourhis, Jean Henri
AU - Mohty, Mohamad
AU - Cornelissen, Jan J
AU - Chevallier, Patrice
AU - Bernasconi, Paolo
AU - Stelljes, Matthias
AU - Rohrlich, Pierre-Simon
AU - Fanin, Renato
AU - Finke, Jürgen
AU - Maertens, Johan
AU - Blaise, Didier
AU - Itälä-Remes, Maija
AU - Labussière-Wallet, Hélène
AU - Robin, Marie
AU - McLornan, Donal
AU - Chalandon, Yves
AU - Yakoub-Agha, Ibrahim
AU - Kröger, Nicolaus
AU - CMWP of the European Society for Blood and Marrow Transplantation
N1 - Copyright © 2019 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.
PY - 2019/6
Y1 - 2019/6
N2 - We aimed to validate the MYelofibrosis SECondary to PV and ET prognostic model (MYSEC-PM) in 159 patients with myelofibrosis secondary to polycythemia vera (PV) and essential thrombocythemia (ET) from the European Society for Blood and Marrow Transplantation registry undergoing transplantation from matched siblings or unrelated donors. Furthermore, we aimed to test its prognostic performance in comparison with the Dynamic International Prognostic Scoring System (DIPSS). Score performance was analyzed using the concordance index (C): the probability that a patient who experienced an event had a higher risk score than a patient who did not (C > .5 suggesting predictive ability). Median follow-up of the total cohort was 41 months (range, 34 to 54), 45 months in post-PV and 38 months in post-ET myelofibrosis. Survival at 1, 2, and 4 years was 70% (95% CI, 63% to 77%), 61% (95% CI, 53% to 69%), and 52% (95% CI, 43% to 61%) for the total cohort; 70% (95% CI, 59% to 80%), 61% (95% CI, 49% to 73%), and 51% (95% CI, 38% to 64%) for post-PV; and 71% (95% CI, 61% to 81%), 61% (95% CI, 50% to 72%), and 54% (95% CI, 42% to 66%) for post-ET myelofibrosis (P = .78). Overall, the DIPSS was not significantly predictive of outcome (P = .28). With respect to the MYSEC-PM, overall survival at 4 years was 69% for the low-risk, 55% for the intermediate 1-risk, 47% for the intermediate 2-risk, and 22% (0% to 45%) for the high-risk groups. The prognostic model was predictive of survival overall (P = .05), whereas groups with intermediate 2 and high risk showed no significant difference (P = .44). Assessment of prognostic utility yielded a C-index of .575 (95% CI, .502 to .648) for the DIPSS, whereas assessment of the MYSEC-PM resulted in a C-statistics of .636 (95% CI, .563 to .708), indicating improvement in prediction of post-transplant survival using the new MYSEC-PM. In addition, transplantations from an unrelated donor in comparison with an HLA-identical sibling showed worse outcome (P = .04), and transplant recipients seropositive for cytomegalovirus in comparison with seronegative recipients (P = .01) showed worse survival. In conclusion, incorporating transplant-specific and clinical and mutational information together with the MYSEC-PM may enhance risk stratification.
AB - We aimed to validate the MYelofibrosis SECondary to PV and ET prognostic model (MYSEC-PM) in 159 patients with myelofibrosis secondary to polycythemia vera (PV) and essential thrombocythemia (ET) from the European Society for Blood and Marrow Transplantation registry undergoing transplantation from matched siblings or unrelated donors. Furthermore, we aimed to test its prognostic performance in comparison with the Dynamic International Prognostic Scoring System (DIPSS). Score performance was analyzed using the concordance index (C): the probability that a patient who experienced an event had a higher risk score than a patient who did not (C > .5 suggesting predictive ability). Median follow-up of the total cohort was 41 months (range, 34 to 54), 45 months in post-PV and 38 months in post-ET myelofibrosis. Survival at 1, 2, and 4 years was 70% (95% CI, 63% to 77%), 61% (95% CI, 53% to 69%), and 52% (95% CI, 43% to 61%) for the total cohort; 70% (95% CI, 59% to 80%), 61% (95% CI, 49% to 73%), and 51% (95% CI, 38% to 64%) for post-PV; and 71% (95% CI, 61% to 81%), 61% (95% CI, 50% to 72%), and 54% (95% CI, 42% to 66%) for post-ET myelofibrosis (P = .78). Overall, the DIPSS was not significantly predictive of outcome (P = .28). With respect to the MYSEC-PM, overall survival at 4 years was 69% for the low-risk, 55% for the intermediate 1-risk, 47% for the intermediate 2-risk, and 22% (0% to 45%) for the high-risk groups. The prognostic model was predictive of survival overall (P = .05), whereas groups with intermediate 2 and high risk showed no significant difference (P = .44). Assessment of prognostic utility yielded a C-index of .575 (95% CI, .502 to .648) for the DIPSS, whereas assessment of the MYSEC-PM resulted in a C-statistics of .636 (95% CI, .563 to .708), indicating improvement in prediction of post-transplant survival using the new MYSEC-PM. In addition, transplantations from an unrelated donor in comparison with an HLA-identical sibling showed worse outcome (P = .04), and transplant recipients seropositive for cytomegalovirus in comparison with seronegative recipients (P = .01) showed worse survival. In conclusion, incorporating transplant-specific and clinical and mutational information together with the MYSEC-PM may enhance risk stratification.
U2 - 10.1016/j.bbmt.2019.03.024
DO - 10.1016/j.bbmt.2019.03.024
M3 - SCORING: Journal article
C2 - 30930192
VL - 25
SP - e204-e208
JO - BIOL BLOOD MARROW TR
JF - BIOL BLOOD MARROW TR
SN - 1083-8791
IS - 6
ER -