A multicenter retrospective evaluation of Chronic Myeloid Leukemia (CML) therapy in Austria assessing the impact of early treatment response on patient outcomes in a real-life setting: R-EFECT study

Andreas L Petzer; Wolfgang R Sperr; Veronika Buxhofer-Ausch; Thamer Sliwa; Stefan Schmidt; Richard Greil; Albert Wölfler; Petra Pichler; Clemens Dormann; Sonja Burgstaller; Christoph Tinchon; Alois Lang; Florian Goebel; Shanow Uthman; Niklas Muenchmeier; Peter Valent

doi:10.1007/s00508-020-01690-1

A multicenter retrospective evaluation of Chronic Myeloid Leukemia (CML) therapy in Austria assessing the impact of early treatment response on patient outcomes in a real-life setting: R-EFECT study

Andreas L Petzer, Wolfgang R Sperr, Veronika Buxhofer-Ausch, Thamer Sliwa, Stefan Schmidt, Richard Greil, Albert Wölfler, Petra Pichler, Clemens Dormann, Sonja Burgstaller, Christoph Tinchon, Alois Lang, Florian Goebel, Shanow Uthman, Niklas Muenchmeier, Peter Valent

Division of Internal Medicine 1 (University Hospital St. Pölten)

Research output: Journal article (peer-reviewed) › Journal article

Abstract

BACKGROUND: Several clinical trials in chronic phase (CP) chronic myeloid leukemia (CML) showed that early response to tyrosine kinase inhibitor (TKI) treatment results in an improved long-term survival and progression-free survival. This study assessed whether patients achieving early treatment response (ETR; partial cytogenetic response or BCR-ABL1 mRNA ≤10% at 3 months) in daily practice also have a long-term survival benefit.

METHODS: The Retrospective Evaluation of Early response in CML for long-term Treatment outcome (R-EFECT), a multicenter, retrospective chart review, documented patients with newly diagnosed CML-CP starting first-line TKI therapy in routine clinical practice. The primary aim was to assess the 5‑year overall survival rate.

RESULTS: Of the 211 patients from 12 centers across Austria (January 2004-May 2010), 176 (median age, 56 years) were included in the analysis. All patients received first-line therapy with imatinib. Overall, 136 patients (77.3%) achieved ETR (ETR+ group), whereas 40 (22.7%) did not reach ETR (ETR- group). The ETR+ group had higher 5‑year overall survival (92.5% vs. 77.5%, P = 0.018) and progression-free survival (95.6% vs. 87.5%, P = 0.06) rates compared with the ETR- group. As expected, more patients in the ETR- group were switched to another TKI. At the last contact, 120 patients were still on imatinib and 44 had switched to another TKI (25 to nilotinib, 15 to dasatinib, and 4 to bosutinib).

CONCLUSION: The data are in line with randomized trials demonstrating that ETR is associated with improved survival and thus confirmed these results in patients treated in daily clinical routine.

Original language	English
Pages (from-to)	415-422
Number of pages	8
Journal	Wiener Klinische Wochenschrift
Volume	132
Issue number	15-16
DOIs	https://doi.org/10.1007/s00508-020-01690-1
Publication status	Published - 1 Aug 2020

Keywords

Adolescent
Adult
Aged
Aged, 80 and over
Antineoplastic Agents/therapeutic use
Austria
Dasatinib/therapeutic use
Female
Humans
Leukemia, Myelogenous, Chronic, BCR-ABL Positive/diagnosis
Leukemia, Myeloid, Chronic-Phase/drug therapy
Male
Middle Aged
Retrospective Studies
Treatment Outcome
Young Adult

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10.1007/s00508-020-01690-1

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Petzer, A. L., Sperr, W. R., Buxhofer-Ausch, V., Sliwa, T., Schmidt, S., Greil, R., Wölfler, A., Pichler, P., Dormann, C., Burgstaller, S., Tinchon, C., Lang, A., Goebel, F., Uthman, S., Muenchmeier, N., & Valent, P. (2020). A multicenter retrospective evaluation of Chronic Myeloid Leukemia (CML) therapy in Austria assessing the impact of early treatment response on patient outcomes in a real-life setting: R-EFECT study. Wiener Klinische Wochenschrift, 132(15-16), 415-422. https://doi.org/10.1007/s00508-020-01690-1

@article{091f83189e5441dc997f9494907406cb,

title = "A multicenter retrospective evaluation of Chronic Myeloid Leukemia (CML) therapy in Austria assessing the impact of early treatment response on patient outcomes in a real-life setting: R-EFECT study",

abstract = "BACKGROUND: Several clinical trials in chronic phase (CP) chronic myeloid leukemia (CML) showed that early response to tyrosine kinase inhibitor (TKI) treatment results in an improved long-term survival and progression-free survival. This study assessed whether patients achieving early treatment response (ETR; partial cytogenetic response or BCR-ABL1 mRNA ≤10% at 3 months) in daily practice also have a long-term survival benefit.METHODS: The Retrospective Evaluation of Early response in CML for long-term Treatment outcome (R-EFECT), a multicenter, retrospective chart review, documented patients with newly diagnosed CML-CP starting first-line TKI therapy in routine clinical practice. The primary aim was to assess the 5‑year overall survival rate.RESULTS: Of the 211 patients from 12 centers across Austria (January 2004-May 2010), 176 (median age, 56 years) were included in the analysis. All patients received first-line therapy with imatinib. Overall, 136 patients (77.3%) achieved ETR (ETR+ group), whereas 40 (22.7%) did not reach ETR (ETR- group). The ETR+ group had higher 5‑year overall survival (92.5% vs. 77.5%, P = 0.018) and progression-free survival (95.6% vs. 87.5%, P = 0.06) rates compared with the ETR- group. As expected, more patients in the ETR- group were switched to another TKI. At the last contact, 120 patients were still on imatinib and 44 had switched to another TKI (25 to nilotinib, 15 to dasatinib, and 4 to bosutinib).CONCLUSION: The data are in line with randomized trials demonstrating that ETR is associated with improved survival and thus confirmed these results in patients treated in daily clinical routine.",

keywords = "Adolescent, Adult, Aged, Aged, 80 and over, Antineoplastic Agents/therapeutic use, Austria, Dasatinib/therapeutic use, Female, Humans, Leukemia, Myelogenous, Chronic, BCR-ABL Positive/diagnosis, Leukemia, Myeloid, Chronic-Phase/drug therapy, Male, Middle Aged, Retrospective Studies, Treatment Outcome, Young Adult",

author = "Petzer, {Andreas L} and Sperr, {Wolfgang R} and Veronika Buxhofer-Ausch and Thamer Sliwa and Stefan Schmidt and Richard Greil and Albert W{\"o}lfler and Petra Pichler and Clemens Dormann and Sonja Burgstaller and Christoph Tinchon and Alois Lang and Florian Goebel and Shanow Uthman and Niklas Muenchmeier and Peter Valent",

note = "Funding Information: A.L. Petzer – Advisory board member and honoraria recipient of Novartis, BMS, Pfizer, and Incyte. Travel support from BMS and Pfizer. W.R. Sperr – Fee from Novartis and Pfizer. V. Buxhofer-Ausch and T. Sliwa declare that they have no competing interests. S. Schmidt – Consultant of BMS. Research Grant from Incyte/Ariad. Advisory board participation and CML registry of Pfizer. R. Greil – Honoraria recipient, consulting or advisor, research funding, travel support, accommodation and expenses from Celgene, Roche, Merck, Takeda, Astra Zeneca, Novartis, Amgen, BMS, MSD, Sandoz, Abbvie, and Janssen. A. W{\"o}lfler – Honoraria recipient of Novartis, BMS, Pfizer, and Incyte. P. Pichler declare that she has no competing interests. C. Dormann – Advisory board member of Pfizer and Eli Lilly. Travel support from Novartis, Pfizer, Roche, MDS, Angelini, Ipsen, and Celgene. Speaker honoraria recipient of Roche, Bristol-Myers Squibb, MDS, Pierre Fabre, and AstraZeneca. S. Burgstaller – Advisory board member, travel support, and speaker honoraria recipient of Novartis, AOP Orphan, Celgene, and Takeda. C. Tinchon declare that he has no competing interests. A. Lang – Grant from Novartis. F. Goebel, S. Uthman and N. Muenchmeier – Employees of Novartis. P. Valent – Grant from Celgene and Pfizer. Fee from Novartis, Celgene, Pfizer, Incyte, and Deciphera. Funding Information: The authors thank Susanne Herndlhofer for her support and contribution to the study. The authors also thank Vijay Kadasi, M.Sc, Novartis Pvt Ltd, for providing medical editorial assistance. Publisher Copyright: {\textcopyright} 2020, The Author(s).",

year = "2020",

month = aug,

day = "1",

doi = "10.1007/s00508-020-01690-1",

language = "English",

volume = "132",

pages = "415--422",

journal = "Wiener Klinische Wochenschrift",

issn = "0043-5325",

publisher = "Springer-Verlag Wien",

number = "15-16",

}

Petzer, AL, Sperr, WR, Buxhofer-Ausch, V, Sliwa, T, Schmidt, S, Greil, R, Wölfler, A, Pichler, P, Dormann, C, Burgstaller, S, Tinchon, C, Lang, A, Goebel, F, Uthman, S, Muenchmeier, N & Valent, P 2020, 'A multicenter retrospective evaluation of Chronic Myeloid Leukemia (CML) therapy in Austria assessing the impact of early treatment response on patient outcomes in a real-life setting: R-EFECT study', Wiener Klinische Wochenschrift, vol. 132, no. 15-16, pp. 415-422. https://doi.org/10.1007/s00508-020-01690-1

A multicenter retrospective evaluation of Chronic Myeloid Leukemia (CML) therapy in Austria assessing the impact of early treatment response on patient outcomes in a real-life setting: R-EFECT study. / Petzer, Andreas L; Sperr, Wolfgang R; Buxhofer-Ausch, Veronika et al.
In: Wiener Klinische Wochenschrift, Vol. 132, No. 15-16, 01.08.2020, p. 415-422.

Research output: Journal article (peer-reviewed) › Journal article

TY - JOUR

T1 - A multicenter retrospective evaluation of Chronic Myeloid Leukemia (CML) therapy in Austria assessing the impact of early treatment response on patient outcomes in a real-life setting

T2 - R-EFECT study

AU - Petzer, Andreas L

AU - Sperr, Wolfgang R

AU - Buxhofer-Ausch, Veronika

AU - Sliwa, Thamer

AU - Schmidt, Stefan

AU - Greil, Richard

AU - Wölfler, Albert

AU - Pichler, Petra

AU - Dormann, Clemens

AU - Burgstaller, Sonja

AU - Tinchon, Christoph

AU - Lang, Alois

AU - Goebel, Florian

AU - Uthman, Shanow

AU - Muenchmeier, Niklas

AU - Valent, Peter

N1 - Funding Information: A.L. Petzer – Advisory board member and honoraria recipient of Novartis, BMS, Pfizer, and Incyte. Travel support from BMS and Pfizer. W.R. Sperr – Fee from Novartis and Pfizer. V. Buxhofer-Ausch and T. Sliwa declare that they have no competing interests. S. Schmidt – Consultant of BMS. Research Grant from Incyte/Ariad. Advisory board participation and CML registry of Pfizer. R. Greil – Honoraria recipient, consulting or advisor, research funding, travel support, accommodation and expenses from Celgene, Roche, Merck, Takeda, Astra Zeneca, Novartis, Amgen, BMS, MSD, Sandoz, Abbvie, and Janssen. A. Wölfler – Honoraria recipient of Novartis, BMS, Pfizer, and Incyte. P. Pichler declare that she has no competing interests. C. Dormann – Advisory board member of Pfizer and Eli Lilly. Travel support from Novartis, Pfizer, Roche, MDS, Angelini, Ipsen, and Celgene. Speaker honoraria recipient of Roche, Bristol-Myers Squibb, MDS, Pierre Fabre, and AstraZeneca. S. Burgstaller – Advisory board member, travel support, and speaker honoraria recipient of Novartis, AOP Orphan, Celgene, and Takeda. C. Tinchon declare that he has no competing interests. A. Lang – Grant from Novartis. F. Goebel, S. Uthman and N. Muenchmeier – Employees of Novartis. P. Valent – Grant from Celgene and Pfizer. Fee from Novartis, Celgene, Pfizer, Incyte, and Deciphera. Funding Information: The authors thank Susanne Herndlhofer for her support and contribution to the study. The authors also thank Vijay Kadasi, M.Sc, Novartis Pvt Ltd, for providing medical editorial assistance. Publisher Copyright: © 2020, The Author(s).

PY - 2020/8/1

Y1 - 2020/8/1

N2 - BACKGROUND: Several clinical trials in chronic phase (CP) chronic myeloid leukemia (CML) showed that early response to tyrosine kinase inhibitor (TKI) treatment results in an improved long-term survival and progression-free survival. This study assessed whether patients achieving early treatment response (ETR; partial cytogenetic response or BCR-ABL1 mRNA ≤10% at 3 months) in daily practice also have a long-term survival benefit.METHODS: The Retrospective Evaluation of Early response in CML for long-term Treatment outcome (R-EFECT), a multicenter, retrospective chart review, documented patients with newly diagnosed CML-CP starting first-line TKI therapy in routine clinical practice. The primary aim was to assess the 5‑year overall survival rate.RESULTS: Of the 211 patients from 12 centers across Austria (January 2004-May 2010), 176 (median age, 56 years) were included in the analysis. All patients received first-line therapy with imatinib. Overall, 136 patients (77.3%) achieved ETR (ETR+ group), whereas 40 (22.7%) did not reach ETR (ETR- group). The ETR+ group had higher 5‑year overall survival (92.5% vs. 77.5%, P = 0.018) and progression-free survival (95.6% vs. 87.5%, P = 0.06) rates compared with the ETR- group. As expected, more patients in the ETR- group were switched to another TKI. At the last contact, 120 patients were still on imatinib and 44 had switched to another TKI (25 to nilotinib, 15 to dasatinib, and 4 to bosutinib).CONCLUSION: The data are in line with randomized trials demonstrating that ETR is associated with improved survival and thus confirmed these results in patients treated in daily clinical routine.

AB - BACKGROUND: Several clinical trials in chronic phase (CP) chronic myeloid leukemia (CML) showed that early response to tyrosine kinase inhibitor (TKI) treatment results in an improved long-term survival and progression-free survival. This study assessed whether patients achieving early treatment response (ETR; partial cytogenetic response or BCR-ABL1 mRNA ≤10% at 3 months) in daily practice also have a long-term survival benefit.METHODS: The Retrospective Evaluation of Early response in CML for long-term Treatment outcome (R-EFECT), a multicenter, retrospective chart review, documented patients with newly diagnosed CML-CP starting first-line TKI therapy in routine clinical practice. The primary aim was to assess the 5‑year overall survival rate.RESULTS: Of the 211 patients from 12 centers across Austria (January 2004-May 2010), 176 (median age, 56 years) were included in the analysis. All patients received first-line therapy with imatinib. Overall, 136 patients (77.3%) achieved ETR (ETR+ group), whereas 40 (22.7%) did not reach ETR (ETR- group). The ETR+ group had higher 5‑year overall survival (92.5% vs. 77.5%, P = 0.018) and progression-free survival (95.6% vs. 87.5%, P = 0.06) rates compared with the ETR- group. As expected, more patients in the ETR- group were switched to another TKI. At the last contact, 120 patients were still on imatinib and 44 had switched to another TKI (25 to nilotinib, 15 to dasatinib, and 4 to bosutinib).CONCLUSION: The data are in line with randomized trials demonstrating that ETR is associated with improved survival and thus confirmed these results in patients treated in daily clinical routine.

KW - Adolescent

KW - Adult

KW - Aged

KW - Aged, 80 and over

KW - Antineoplastic Agents/therapeutic use

KW - Austria

KW - Dasatinib/therapeutic use

KW - Female

KW - Humans

KW - Leukemia, Myelogenous, Chronic, BCR-ABL Positive/diagnosis

KW - Leukemia, Myeloid, Chronic-Phase/drug therapy

KW - Male

KW - Middle Aged

KW - Retrospective Studies

KW - Treatment Outcome

KW - Young Adult

UR - http://www.scopus.com/inward/record.url?scp=85086275286&partnerID=8YFLogxK

U2 - 10.1007/s00508-020-01690-1

DO - 10.1007/s00508-020-01690-1

M3 - Journal article

C2 - 32533444

SN - 0043-5325

VL - 132

SP - 415

EP - 422

JO - Wiener Klinische Wochenschrift

JF - Wiener Klinische Wochenschrift

IS - 15-16

ER -

Petzer AL, Sperr WR, Buxhofer-Ausch V, Sliwa T, Schmidt S, Greil R et al. A multicenter retrospective evaluation of Chronic Myeloid Leukemia (CML) therapy in Austria assessing the impact of early treatment response on patient outcomes in a real-life setting: R-EFECT study. Wiener Klinische Wochenschrift. 2020 Aug 1;132(15-16):415-422. doi: 10.1007/s00508-020-01690-1

A multicenter retrospective evaluation of Chronic Myeloid Leukemia (CML) therapy in Austria assessing the impact of early treatment response on patient outcomes in a real-life setting: R-EFECT study

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Keywords

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