Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis : myelodysplastic syndrome
Advanced systemic mastocytosis (AdvSM) is a rare myeloid neoplasm associated with poor overall survival (OS). This study (NCT04695431) compared clinical outcomes between patients with AdvSM treated with avapritinib in the Phase 1 EXPLORER (NCT0256198) and Phase 2 PATHFINDER (NCT03580655) trials (N =...
Ausführliche Beschreibung
Autor*in: |
Reiter, Andreas - 1964- [verfasserIn] Gotlib, Jason [verfasserIn] Álvarez-Twose, Iván [verfasserIn] Radia, Deepti H. [verfasserIn] Lübke, Johannes - 1996- [verfasserIn] Bobbili, Priyanka J. [verfasserIn] Wang, Aolin [verfasserIn] Norregaard, Chelsea [verfasserIn] Dimitrijevic, Saša [verfasserIn] Sullivan, Erin [verfasserIn] Louie-Gao, Melinda [verfasserIn] Schwaab, Juliana - 1982- [verfasserIn] Galinsky, Ilene A. [verfasserIn] Perkins, Cecelia [verfasserIn] Sperr, Wolfgang R. [verfasserIn] Sriskandarajah, Priya [verfasserIn] Chin, Andi [verfasserIn] Sendhil, Selvam R. [verfasserIn] Duh, Mei Sheng [verfasserIn] Valent, Peter [verfasserIn] DeAngelo, Daniel J. [verfasserIn] |
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E-Artikel |
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Sprache: |
Englisch |
Erschienen: |
05 July 2022 |
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Schlagwörter: |
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Anmerkung: |
Gesehen am 31.10.2023 |
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Umfang: |
13 |
Übergeordnetes Werk: |
Enthalten in: Leukemia - London : Springer Nature, 1997, 36(2022), 8, Seite 2108-2120 |
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Übergeordnetes Werk: |
volume:36 ; year:2022 ; number:8 ; pages:2108-2120 ; extent:13 |
Links: |
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DOI / URN: |
10.1038/s41375-022-01615-z |
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Katalog-ID: |
1868839656 |
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245 | 1 | 0 | |a Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis |b myelodysplastic syndrome |c Andreas Reiter, Jason Gotlib, Iván Álvarez-Twose, Deepti H. Radia, Johannes Lübke, Priyanka J. Bobbili, Aolin Wang, Chelsea Norregaard, Saša Dimitrijevic, Erin Sullivan, Melinda Louie-Gao, Juliana Schwaab, Ilene A. Galinsky, Cecelia Perkins, Wolfgang R. Sperr, Priya Sriskandarajah, Andi Chin, Selvam R. Sendhil, Mei Sheng Duh, Peter Valent and Daniel J. DeAngelo |
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520 | |a Advanced systemic mastocytosis (AdvSM) is a rare myeloid neoplasm associated with poor overall survival (OS). This study (NCT04695431) compared clinical outcomes between patients with AdvSM treated with avapritinib in the Phase 1 EXPLORER (NCT0256198) and Phase 2 PATHFINDER (NCT03580655) trials (N = 176) and patients treated with best available therapy (BAT; N = 141). A multi-center, observational, retrospective chart review study was conducted at six study sites (four European, two American) to collect data from patients with AdvSM who received BAT; these data were pooled with data from EXPLORER and PATHFINDER. Comparisons between outcomes of OS, duration of treatment (DOT), and maximum reduction in serum tryptase were conducted between the treatment cohorts, with adjustment for key covariates. The results indicated that the avapritinib cohort had significantly better survival (adjusted hazard ratio (HR) (95% confidence interval (CI)): 0.48 (0.29, 0.79); p = 0.004) and significantly longer DOT (HR: 0.36 (0.26, 0.51); p < 0.001) compared to the BAT cohort. Additionally, the mean difference in percentage maximum reduction in serum tryptase levels was 60.3% greater in the avapritinib cohort (95% CI: −72.8, −47.9; p < 0.001). With no randomized controlled trials comparing avapritinib to BAT, these data offer crucial insights into the improved efficacy of avapritinib for the treatment of AdvSM. | ||
650 | 4 | |a Cancer therapy | |
650 | 4 | |a Targeted therapies | |
700 | 1 | |a Gotlib, Jason |e verfasserin |4 aut | |
700 | 1 | |a Álvarez-Twose, Iván |e verfasserin |4 aut | |
700 | 1 | |a Radia, Deepti H. |e verfasserin |4 aut | |
700 | 1 | |a Lübke, Johannes |d 1996- |e verfasserin |0 (DE-588)1189769867 |0 (DE-627)1668483645 |4 aut | |
700 | 1 | |a Bobbili, Priyanka J. |e verfasserin |4 aut | |
700 | 1 | |a Wang, Aolin |e verfasserin |4 aut | |
700 | 1 | |a Norregaard, Chelsea |e verfasserin |4 aut | |
700 | 1 | |a Dimitrijevic, Saša |e verfasserin |4 aut | |
700 | 1 | |a Sullivan, Erin |e verfasserin |4 aut | |
700 | 1 | |a Louie-Gao, Melinda |e verfasserin |4 aut | |
700 | 1 | |a Schwaab, Juliana |d 1982- |e verfasserin |0 (DE-588)1051912687 |0 (DE-627)787019399 |0 (DE-576)407558764 |4 aut | |
700 | 1 | |a Galinsky, Ilene A. |e verfasserin |4 aut | |
700 | 1 | |a Perkins, Cecelia |e verfasserin |4 aut | |
700 | 1 | |a Sperr, Wolfgang R. |e verfasserin |4 aut | |
700 | 1 | |a Sriskandarajah, Priya |e verfasserin |4 aut | |
700 | 1 | |a Chin, Andi |e verfasserin |4 aut | |
700 | 1 | |a Sendhil, Selvam R. |e verfasserin |4 aut | |
700 | 1 | |a Duh, Mei Sheng |e verfasserin |4 aut | |
700 | 1 | |a Valent, Peter |e verfasserin |4 aut | |
700 | 1 | |a DeAngelo, Daniel J. |e verfasserin |4 aut | |
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05 July 2022 |
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2022 |
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10.1038/s41375-022-01615-z doi (DE-627)1868839656 (DE-599)KXP1868839656 (OCoLC)1425216584 DE-627 ger DE-627 rda eng Reiter, Andreas 1964- verfasserin (DE-588)172629551 (DE-627)697563766 (DE-576)133488594 aut Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis myelodysplastic syndrome Andreas Reiter, Jason Gotlib, Iván Álvarez-Twose, Deepti H. Radia, Johannes Lübke, Priyanka J. Bobbili, Aolin Wang, Chelsea Norregaard, Saša Dimitrijevic, Erin Sullivan, Melinda Louie-Gao, Juliana Schwaab, Ilene A. Galinsky, Cecelia Perkins, Wolfgang R. Sperr, Priya Sriskandarajah, Andi Chin, Selvam R. Sendhil, Mei Sheng Duh, Peter Valent and Daniel J. DeAngelo 05 July 2022 13 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Gesehen am 31.10.2023 Advanced systemic mastocytosis (AdvSM) is a rare myeloid neoplasm associated with poor overall survival (OS). This study (NCT04695431) compared clinical outcomes between patients with AdvSM treated with avapritinib in the Phase 1 EXPLORER (NCT0256198) and Phase 2 PATHFINDER (NCT03580655) trials (N = 176) and patients treated with best available therapy (BAT; N = 141). A multi-center, observational, retrospective chart review study was conducted at six study sites (four European, two American) to collect data from patients with AdvSM who received BAT; these data were pooled with data from EXPLORER and PATHFINDER. Comparisons between outcomes of OS, duration of treatment (DOT), and maximum reduction in serum tryptase were conducted between the treatment cohorts, with adjustment for key covariates. The results indicated that the avapritinib cohort had significantly better survival (adjusted hazard ratio (HR) (95% confidence interval (CI)): 0.48 (0.29, 0.79); p = 0.004) and significantly longer DOT (HR: 0.36 (0.26, 0.51); p < 0.001) compared to the BAT cohort. Additionally, the mean difference in percentage maximum reduction in serum tryptase levels was 60.3% greater in the avapritinib cohort (95% CI: −72.8, −47.9; p < 0.001). With no randomized controlled trials comparing avapritinib to BAT, these data offer crucial insights into the improved efficacy of avapritinib for the treatment of AdvSM. Cancer therapy Targeted therapies Gotlib, Jason verfasserin aut Álvarez-Twose, Iván verfasserin aut Radia, Deepti H. verfasserin aut Lübke, Johannes 1996- verfasserin (DE-588)1189769867 (DE-627)1668483645 aut Bobbili, Priyanka J. verfasserin aut Wang, Aolin verfasserin aut Norregaard, Chelsea verfasserin aut Dimitrijevic, Saša verfasserin aut Sullivan, Erin verfasserin aut Louie-Gao, Melinda verfasserin aut Schwaab, Juliana 1982- verfasserin (DE-588)1051912687 (DE-627)787019399 (DE-576)407558764 aut Galinsky, Ilene A. verfasserin aut Perkins, Cecelia verfasserin aut Sperr, Wolfgang R. verfasserin aut Sriskandarajah, Priya verfasserin aut Chin, Andi verfasserin aut Sendhil, Selvam R. verfasserin aut Duh, Mei Sheng verfasserin aut Valent, Peter verfasserin aut DeAngelo, Daniel J. verfasserin aut Enthalten in Leukemia London : Springer Nature, 1997 36(2022), 8, Seite 2108-2120 Online-Ressource (DE-627)32046699X (DE-600)2008023-2 (DE-576)094139733 1476-5551 nnns volume:36 year:2022 number:8 pages:2108-2120 extent:13 https://doi.org/10.1038/s41375-022-01615-z Verlag Resolving-System kostenfrei Volltext http://www.nature.com/articles/s41375-022-01615-z Verlag kostenfrei Volltext GBV_USEFLAG_U GBV_ILN_2013 ISIL_DE-16-250 SYSFLAG_1 GBV_KXP GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_32 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_70 GBV_ILN_73 GBV_ILN_74 GBV_ILN_90 GBV_ILN_95 GBV_ILN_100 GBV_ILN_101 GBV_ILN_105 GBV_ILN_110 GBV_ILN_138 GBV_ILN_150 GBV_ILN_151 GBV_ILN_152 GBV_ILN_161 GBV_ILN_170 GBV_ILN_171 GBV_ILN_187 GBV_ILN_213 GBV_ILN_224 GBV_ILN_230 GBV_ILN_250 GBV_ILN_281 GBV_ILN_285 GBV_ILN_293 GBV_ILN_370 GBV_ILN_602 GBV_ILN_636 GBV_ILN_702 GBV_ILN_2001 GBV_ILN_2003 GBV_ILN_2004 GBV_ILN_2005 GBV_ILN_2006 GBV_ILN_2007 GBV_ILN_2008 GBV_ILN_2009 GBV_ILN_2010 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2015 GBV_ILN_2020 GBV_ILN_2021 GBV_ILN_2025 GBV_ILN_2026 GBV_ILN_2027 GBV_ILN_2031 GBV_ILN_2034 GBV_ILN_2037 GBV_ILN_2038 GBV_ILN_2039 GBV_ILN_2044 GBV_ILN_2048 GBV_ILN_2049 GBV_ILN_2050 GBV_ILN_2055 GBV_ILN_2056 GBV_ILN_2057 GBV_ILN_2059 GBV_ILN_2061 GBV_ILN_2064 GBV_ILN_2065 GBV_ILN_2068 GBV_ILN_2088 GBV_ILN_2093 GBV_ILN_2106 GBV_ILN_2107 GBV_ILN_2108 GBV_ILN_2110 GBV_ILN_2111 GBV_ILN_2112 GBV_ILN_2113 GBV_ILN_2118 GBV_ILN_2122 GBV_ILN_2129 GBV_ILN_2143 GBV_ILN_2144 GBV_ILN_2147 GBV_ILN_2148 GBV_ILN_2152 GBV_ILN_2153 GBV_ILN_2190 GBV_ILN_2232 GBV_ILN_2336 GBV_ILN_2446 GBV_ILN_2470 GBV_ILN_2472 GBV_ILN_2507 GBV_ILN_2522 GBV_ILN_4012 GBV_ILN_4035 GBV_ILN_4037 GBV_ILN_4046 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4242 GBV_ILN_4246 GBV_ILN_4249 GBV_ILN_4251 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4326 GBV_ILN_4328 GBV_ILN_4333 GBV_ILN_4334 GBV_ILN_4335 GBV_ILN_4336 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4393 GBV_ILN_4700 GBV_ILN_2403 GBV_ILN_2403 ISIL_DE-LFER AR 36 2022 8 2108-2120 13 2013 01 DE-16-250 4400302644 00 --%%-- --%%-- --%%-- --%%-- l01 31-10-23 2403 01 DE-LFER 4409565559 00 --%%-- --%%-- n --%%-- l01 16-11-23 2403 01 DE-LFER https://doi.org/10.1038/s41375-022-01615-z 2403 01 DE-LFER http://www.nature.com/articles/s41375-022-01615-z 2013 01 DE-16-250 00 s hd2022 2013 01 DE-16-250 01 s (DE-627)1410508463 wissenschaftlicher Artikel (Zeitschrift) 2013 01 DE-16-250 02 s per_21 2013 01 DE-16-250 03 s s_13 2013 01 DE-16-250 04 p (DE-627)1446321282 Reiter, Andreas 2013 01 DE-16-250 04 k (DE-627)1416468528 III. Medizinische Klinik 2013 01 DE-16-250 04 s (DE-627)1410501914 Verfasser 2013 01 DE-16-250 04 s pos_1 2013 01 DE-16-250 05 p (DE-627)1668484099 Lübke, Johannes 2013 01 DE-16-250 05 k (DE-627)1416468528 III. Medizinische Klinik 2013 01 DE-16-250 05 s (DE-627)1410501914 Verfasser 2013 01 DE-16-250 05 s pos_5 2013 01 DE-16-250 06 p (DE-627)1477558896 Schwaab, Juliana 2013 01 DE-16-250 06 k (DE-627)1416468528 III. Medizinische Klinik 2013 01 DE-16-250 06 s (DE-627)1410501914 Verfasser 2013 01 DE-16-250 06 s pos_12 |
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10.1038/s41375-022-01615-z doi (DE-627)1868839656 (DE-599)KXP1868839656 (OCoLC)1425216584 DE-627 ger DE-627 rda eng Reiter, Andreas 1964- verfasserin (DE-588)172629551 (DE-627)697563766 (DE-576)133488594 aut Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis myelodysplastic syndrome Andreas Reiter, Jason Gotlib, Iván Álvarez-Twose, Deepti H. Radia, Johannes Lübke, Priyanka J. Bobbili, Aolin Wang, Chelsea Norregaard, Saša Dimitrijevic, Erin Sullivan, Melinda Louie-Gao, Juliana Schwaab, Ilene A. Galinsky, Cecelia Perkins, Wolfgang R. Sperr, Priya Sriskandarajah, Andi Chin, Selvam R. Sendhil, Mei Sheng Duh, Peter Valent and Daniel J. DeAngelo 05 July 2022 13 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Gesehen am 31.10.2023 Advanced systemic mastocytosis (AdvSM) is a rare myeloid neoplasm associated with poor overall survival (OS). This study (NCT04695431) compared clinical outcomes between patients with AdvSM treated with avapritinib in the Phase 1 EXPLORER (NCT0256198) and Phase 2 PATHFINDER (NCT03580655) trials (N = 176) and patients treated with best available therapy (BAT; N = 141). A multi-center, observational, retrospective chart review study was conducted at six study sites (four European, two American) to collect data from patients with AdvSM who received BAT; these data were pooled with data from EXPLORER and PATHFINDER. Comparisons between outcomes of OS, duration of treatment (DOT), and maximum reduction in serum tryptase were conducted between the treatment cohorts, with adjustment for key covariates. The results indicated that the avapritinib cohort had significantly better survival (adjusted hazard ratio (HR) (95% confidence interval (CI)): 0.48 (0.29, 0.79); p = 0.004) and significantly longer DOT (HR: 0.36 (0.26, 0.51); p < 0.001) compared to the BAT cohort. Additionally, the mean difference in percentage maximum reduction in serum tryptase levels was 60.3% greater in the avapritinib cohort (95% CI: −72.8, −47.9; p < 0.001). With no randomized controlled trials comparing avapritinib to BAT, these data offer crucial insights into the improved efficacy of avapritinib for the treatment of AdvSM. Cancer therapy Targeted therapies Gotlib, Jason verfasserin aut Álvarez-Twose, Iván verfasserin aut Radia, Deepti H. verfasserin aut Lübke, Johannes 1996- verfasserin (DE-588)1189769867 (DE-627)1668483645 aut Bobbili, Priyanka J. verfasserin aut Wang, Aolin verfasserin aut Norregaard, Chelsea verfasserin aut Dimitrijevic, Saša verfasserin aut Sullivan, Erin verfasserin aut Louie-Gao, Melinda verfasserin aut Schwaab, Juliana 1982- verfasserin (DE-588)1051912687 (DE-627)787019399 (DE-576)407558764 aut Galinsky, Ilene A. verfasserin aut Perkins, Cecelia verfasserin aut Sperr, Wolfgang R. verfasserin aut Sriskandarajah, Priya verfasserin aut Chin, Andi verfasserin aut Sendhil, Selvam R. verfasserin aut Duh, Mei Sheng verfasserin aut Valent, Peter verfasserin aut DeAngelo, Daniel J. verfasserin aut Enthalten in Leukemia London : Springer Nature, 1997 36(2022), 8, Seite 2108-2120 Online-Ressource (DE-627)32046699X (DE-600)2008023-2 (DE-576)094139733 1476-5551 nnns volume:36 year:2022 number:8 pages:2108-2120 extent:13 https://doi.org/10.1038/s41375-022-01615-z Verlag Resolving-System kostenfrei Volltext http://www.nature.com/articles/s41375-022-01615-z Verlag kostenfrei Volltext GBV_USEFLAG_U GBV_ILN_2013 ISIL_DE-16-250 SYSFLAG_1 GBV_KXP GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_32 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_70 GBV_ILN_73 GBV_ILN_74 GBV_ILN_90 GBV_ILN_95 GBV_ILN_100 GBV_ILN_101 GBV_ILN_105 GBV_ILN_110 GBV_ILN_138 GBV_ILN_150 GBV_ILN_151 GBV_ILN_152 GBV_ILN_161 GBV_ILN_170 GBV_ILN_171 GBV_ILN_187 GBV_ILN_213 GBV_ILN_224 GBV_ILN_230 GBV_ILN_250 GBV_ILN_281 GBV_ILN_285 GBV_ILN_293 GBV_ILN_370 GBV_ILN_602 GBV_ILN_636 GBV_ILN_702 GBV_ILN_2001 GBV_ILN_2003 GBV_ILN_2004 GBV_ILN_2005 GBV_ILN_2006 GBV_ILN_2007 GBV_ILN_2008 GBV_ILN_2009 GBV_ILN_2010 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2015 GBV_ILN_2020 GBV_ILN_2021 GBV_ILN_2025 GBV_ILN_2026 GBV_ILN_2027 GBV_ILN_2031 GBV_ILN_2034 GBV_ILN_2037 GBV_ILN_2038 GBV_ILN_2039 GBV_ILN_2044 GBV_ILN_2048 GBV_ILN_2049 GBV_ILN_2050 GBV_ILN_2055 GBV_ILN_2056 GBV_ILN_2057 GBV_ILN_2059 GBV_ILN_2061 GBV_ILN_2064 GBV_ILN_2065 GBV_ILN_2068 GBV_ILN_2088 GBV_ILN_2093 GBV_ILN_2106 GBV_ILN_2107 GBV_ILN_2108 GBV_ILN_2110 GBV_ILN_2111 GBV_ILN_2112 GBV_ILN_2113 GBV_ILN_2118 GBV_ILN_2122 GBV_ILN_2129 GBV_ILN_2143 GBV_ILN_2144 GBV_ILN_2147 GBV_ILN_2148 GBV_ILN_2152 GBV_ILN_2153 GBV_ILN_2190 GBV_ILN_2232 GBV_ILN_2336 GBV_ILN_2446 GBV_ILN_2470 GBV_ILN_2472 GBV_ILN_2507 GBV_ILN_2522 GBV_ILN_4012 GBV_ILN_4035 GBV_ILN_4037 GBV_ILN_4046 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4242 GBV_ILN_4246 GBV_ILN_4249 GBV_ILN_4251 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4326 GBV_ILN_4328 GBV_ILN_4333 GBV_ILN_4334 GBV_ILN_4335 GBV_ILN_4336 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4393 GBV_ILN_4700 GBV_ILN_2403 GBV_ILN_2403 ISIL_DE-LFER AR 36 2022 8 2108-2120 13 2013 01 DE-16-250 4400302644 00 --%%-- --%%-- --%%-- --%%-- l01 31-10-23 2403 01 DE-LFER 4409565559 00 --%%-- --%%-- n --%%-- l01 16-11-23 2403 01 DE-LFER https://doi.org/10.1038/s41375-022-01615-z 2403 01 DE-LFER http://www.nature.com/articles/s41375-022-01615-z 2013 01 DE-16-250 00 s hd2022 2013 01 DE-16-250 01 s (DE-627)1410508463 wissenschaftlicher Artikel (Zeitschrift) 2013 01 DE-16-250 02 s per_21 2013 01 DE-16-250 03 s s_13 2013 01 DE-16-250 04 p (DE-627)1446321282 Reiter, Andreas 2013 01 DE-16-250 04 k (DE-627)1416468528 III. Medizinische Klinik 2013 01 DE-16-250 04 s (DE-627)1410501914 Verfasser 2013 01 DE-16-250 04 s pos_1 2013 01 DE-16-250 05 p (DE-627)1668484099 Lübke, Johannes 2013 01 DE-16-250 05 k (DE-627)1416468528 III. Medizinische Klinik 2013 01 DE-16-250 05 s (DE-627)1410501914 Verfasser 2013 01 DE-16-250 05 s pos_5 2013 01 DE-16-250 06 p (DE-627)1477558896 Schwaab, Juliana 2013 01 DE-16-250 06 k (DE-627)1416468528 III. Medizinische Klinik 2013 01 DE-16-250 06 s (DE-627)1410501914 Verfasser 2013 01 DE-16-250 06 s pos_12 |
allfields_unstemmed |
10.1038/s41375-022-01615-z doi (DE-627)1868839656 (DE-599)KXP1868839656 (OCoLC)1425216584 DE-627 ger DE-627 rda eng Reiter, Andreas 1964- verfasserin (DE-588)172629551 (DE-627)697563766 (DE-576)133488594 aut Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis myelodysplastic syndrome Andreas Reiter, Jason Gotlib, Iván Álvarez-Twose, Deepti H. Radia, Johannes Lübke, Priyanka J. Bobbili, Aolin Wang, Chelsea Norregaard, Saša Dimitrijevic, Erin Sullivan, Melinda Louie-Gao, Juliana Schwaab, Ilene A. Galinsky, Cecelia Perkins, Wolfgang R. Sperr, Priya Sriskandarajah, Andi Chin, Selvam R. Sendhil, Mei Sheng Duh, Peter Valent and Daniel J. DeAngelo 05 July 2022 13 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Gesehen am 31.10.2023 Advanced systemic mastocytosis (AdvSM) is a rare myeloid neoplasm associated with poor overall survival (OS). This study (NCT04695431) compared clinical outcomes between patients with AdvSM treated with avapritinib in the Phase 1 EXPLORER (NCT0256198) and Phase 2 PATHFINDER (NCT03580655) trials (N = 176) and patients treated with best available therapy (BAT; N = 141). A multi-center, observational, retrospective chart review study was conducted at six study sites (four European, two American) to collect data from patients with AdvSM who received BAT; these data were pooled with data from EXPLORER and PATHFINDER. Comparisons between outcomes of OS, duration of treatment (DOT), and maximum reduction in serum tryptase were conducted between the treatment cohorts, with adjustment for key covariates. The results indicated that the avapritinib cohort had significantly better survival (adjusted hazard ratio (HR) (95% confidence interval (CI)): 0.48 (0.29, 0.79); p = 0.004) and significantly longer DOT (HR: 0.36 (0.26, 0.51); p < 0.001) compared to the BAT cohort. Additionally, the mean difference in percentage maximum reduction in serum tryptase levels was 60.3% greater in the avapritinib cohort (95% CI: −72.8, −47.9; p < 0.001). With no randomized controlled trials comparing avapritinib to BAT, these data offer crucial insights into the improved efficacy of avapritinib for the treatment of AdvSM. Cancer therapy Targeted therapies Gotlib, Jason verfasserin aut Álvarez-Twose, Iván verfasserin aut Radia, Deepti H. verfasserin aut Lübke, Johannes 1996- verfasserin (DE-588)1189769867 (DE-627)1668483645 aut Bobbili, Priyanka J. verfasserin aut Wang, Aolin verfasserin aut Norregaard, Chelsea verfasserin aut Dimitrijevic, Saša verfasserin aut Sullivan, Erin verfasserin aut Louie-Gao, Melinda verfasserin aut Schwaab, Juliana 1982- verfasserin (DE-588)1051912687 (DE-627)787019399 (DE-576)407558764 aut Galinsky, Ilene A. verfasserin aut Perkins, Cecelia verfasserin aut Sperr, Wolfgang R. verfasserin aut Sriskandarajah, Priya verfasserin aut Chin, Andi verfasserin aut Sendhil, Selvam R. verfasserin aut Duh, Mei Sheng verfasserin aut Valent, Peter verfasserin aut DeAngelo, Daniel J. verfasserin aut Enthalten in Leukemia London : Springer Nature, 1997 36(2022), 8, Seite 2108-2120 Online-Ressource (DE-627)32046699X (DE-600)2008023-2 (DE-576)094139733 1476-5551 nnns volume:36 year:2022 number:8 pages:2108-2120 extent:13 https://doi.org/10.1038/s41375-022-01615-z Verlag Resolving-System kostenfrei Volltext http://www.nature.com/articles/s41375-022-01615-z Verlag kostenfrei Volltext GBV_USEFLAG_U GBV_ILN_2013 ISIL_DE-16-250 SYSFLAG_1 GBV_KXP GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_32 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_70 GBV_ILN_73 GBV_ILN_74 GBV_ILN_90 GBV_ILN_95 GBV_ILN_100 GBV_ILN_101 GBV_ILN_105 GBV_ILN_110 GBV_ILN_138 GBV_ILN_150 GBV_ILN_151 GBV_ILN_152 GBV_ILN_161 GBV_ILN_170 GBV_ILN_171 GBV_ILN_187 GBV_ILN_213 GBV_ILN_224 GBV_ILN_230 GBV_ILN_250 GBV_ILN_281 GBV_ILN_285 GBV_ILN_293 GBV_ILN_370 GBV_ILN_602 GBV_ILN_636 GBV_ILN_702 GBV_ILN_2001 GBV_ILN_2003 GBV_ILN_2004 GBV_ILN_2005 GBV_ILN_2006 GBV_ILN_2007 GBV_ILN_2008 GBV_ILN_2009 GBV_ILN_2010 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2015 GBV_ILN_2020 GBV_ILN_2021 GBV_ILN_2025 GBV_ILN_2026 GBV_ILN_2027 GBV_ILN_2031 GBV_ILN_2034 GBV_ILN_2037 GBV_ILN_2038 GBV_ILN_2039 GBV_ILN_2044 GBV_ILN_2048 GBV_ILN_2049 GBV_ILN_2050 GBV_ILN_2055 GBV_ILN_2056 GBV_ILN_2057 GBV_ILN_2059 GBV_ILN_2061 GBV_ILN_2064 GBV_ILN_2065 GBV_ILN_2068 GBV_ILN_2088 GBV_ILN_2093 GBV_ILN_2106 GBV_ILN_2107 GBV_ILN_2108 GBV_ILN_2110 GBV_ILN_2111 GBV_ILN_2112 GBV_ILN_2113 GBV_ILN_2118 GBV_ILN_2122 GBV_ILN_2129 GBV_ILN_2143 GBV_ILN_2144 GBV_ILN_2147 GBV_ILN_2148 GBV_ILN_2152 GBV_ILN_2153 GBV_ILN_2190 GBV_ILN_2232 GBV_ILN_2336 GBV_ILN_2446 GBV_ILN_2470 GBV_ILN_2472 GBV_ILN_2507 GBV_ILN_2522 GBV_ILN_4012 GBV_ILN_4035 GBV_ILN_4037 GBV_ILN_4046 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4242 GBV_ILN_4246 GBV_ILN_4249 GBV_ILN_4251 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4326 GBV_ILN_4328 GBV_ILN_4333 GBV_ILN_4334 GBV_ILN_4335 GBV_ILN_4336 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4393 GBV_ILN_4700 GBV_ILN_2403 GBV_ILN_2403 ISIL_DE-LFER AR 36 2022 8 2108-2120 13 2013 01 DE-16-250 4400302644 00 --%%-- --%%-- --%%-- --%%-- l01 31-10-23 2403 01 DE-LFER 4409565559 00 --%%-- --%%-- n --%%-- l01 16-11-23 2403 01 DE-LFER https://doi.org/10.1038/s41375-022-01615-z 2403 01 DE-LFER http://www.nature.com/articles/s41375-022-01615-z 2013 01 DE-16-250 00 s hd2022 2013 01 DE-16-250 01 s (DE-627)1410508463 wissenschaftlicher Artikel (Zeitschrift) 2013 01 DE-16-250 02 s per_21 2013 01 DE-16-250 03 s s_13 2013 01 DE-16-250 04 p (DE-627)1446321282 Reiter, Andreas 2013 01 DE-16-250 04 k (DE-627)1416468528 III. Medizinische Klinik 2013 01 DE-16-250 04 s (DE-627)1410501914 Verfasser 2013 01 DE-16-250 04 s pos_1 2013 01 DE-16-250 05 p (DE-627)1668484099 Lübke, Johannes 2013 01 DE-16-250 05 k (DE-627)1416468528 III. Medizinische Klinik 2013 01 DE-16-250 05 s (DE-627)1410501914 Verfasser 2013 01 DE-16-250 05 s pos_5 2013 01 DE-16-250 06 p (DE-627)1477558896 Schwaab, Juliana 2013 01 DE-16-250 06 k (DE-627)1416468528 III. Medizinische Klinik 2013 01 DE-16-250 06 s (DE-627)1410501914 Verfasser 2013 01 DE-16-250 06 s pos_12 |
allfieldsGer |
10.1038/s41375-022-01615-z doi (DE-627)1868839656 (DE-599)KXP1868839656 (OCoLC)1425216584 DE-627 ger DE-627 rda eng Reiter, Andreas 1964- verfasserin (DE-588)172629551 (DE-627)697563766 (DE-576)133488594 aut Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis myelodysplastic syndrome Andreas Reiter, Jason Gotlib, Iván Álvarez-Twose, Deepti H. Radia, Johannes Lübke, Priyanka J. Bobbili, Aolin Wang, Chelsea Norregaard, Saša Dimitrijevic, Erin Sullivan, Melinda Louie-Gao, Juliana Schwaab, Ilene A. Galinsky, Cecelia Perkins, Wolfgang R. Sperr, Priya Sriskandarajah, Andi Chin, Selvam R. Sendhil, Mei Sheng Duh, Peter Valent and Daniel J. DeAngelo 05 July 2022 13 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Gesehen am 31.10.2023 Advanced systemic mastocytosis (AdvSM) is a rare myeloid neoplasm associated with poor overall survival (OS). This study (NCT04695431) compared clinical outcomes between patients with AdvSM treated with avapritinib in the Phase 1 EXPLORER (NCT0256198) and Phase 2 PATHFINDER (NCT03580655) trials (N = 176) and patients treated with best available therapy (BAT; N = 141). A multi-center, observational, retrospective chart review study was conducted at six study sites (four European, two American) to collect data from patients with AdvSM who received BAT; these data were pooled with data from EXPLORER and PATHFINDER. Comparisons between outcomes of OS, duration of treatment (DOT), and maximum reduction in serum tryptase were conducted between the treatment cohorts, with adjustment for key covariates. The results indicated that the avapritinib cohort had significantly better survival (adjusted hazard ratio (HR) (95% confidence interval (CI)): 0.48 (0.29, 0.79); p = 0.004) and significantly longer DOT (HR: 0.36 (0.26, 0.51); p < 0.001) compared to the BAT cohort. Additionally, the mean difference in percentage maximum reduction in serum tryptase levels was 60.3% greater in the avapritinib cohort (95% CI: −72.8, −47.9; p < 0.001). With no randomized controlled trials comparing avapritinib to BAT, these data offer crucial insights into the improved efficacy of avapritinib for the treatment of AdvSM. Cancer therapy Targeted therapies Gotlib, Jason verfasserin aut Álvarez-Twose, Iván verfasserin aut Radia, Deepti H. verfasserin aut Lübke, Johannes 1996- verfasserin (DE-588)1189769867 (DE-627)1668483645 aut Bobbili, Priyanka J. verfasserin aut Wang, Aolin verfasserin aut Norregaard, Chelsea verfasserin aut Dimitrijevic, Saša verfasserin aut Sullivan, Erin verfasserin aut Louie-Gao, Melinda verfasserin aut Schwaab, Juliana 1982- verfasserin (DE-588)1051912687 (DE-627)787019399 (DE-576)407558764 aut Galinsky, Ilene A. verfasserin aut Perkins, Cecelia verfasserin aut Sperr, Wolfgang R. verfasserin aut Sriskandarajah, Priya verfasserin aut Chin, Andi verfasserin aut Sendhil, Selvam R. verfasserin aut Duh, Mei Sheng verfasserin aut Valent, Peter verfasserin aut DeAngelo, Daniel J. verfasserin aut Enthalten in Leukemia London : Springer Nature, 1997 36(2022), 8, Seite 2108-2120 Online-Ressource (DE-627)32046699X (DE-600)2008023-2 (DE-576)094139733 1476-5551 nnns volume:36 year:2022 number:8 pages:2108-2120 extent:13 https://doi.org/10.1038/s41375-022-01615-z Verlag Resolving-System kostenfrei Volltext http://www.nature.com/articles/s41375-022-01615-z Verlag kostenfrei Volltext GBV_USEFLAG_U GBV_ILN_2013 ISIL_DE-16-250 SYSFLAG_1 GBV_KXP GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_32 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_70 GBV_ILN_73 GBV_ILN_74 GBV_ILN_90 GBV_ILN_95 GBV_ILN_100 GBV_ILN_101 GBV_ILN_105 GBV_ILN_110 GBV_ILN_138 GBV_ILN_150 GBV_ILN_151 GBV_ILN_152 GBV_ILN_161 GBV_ILN_170 GBV_ILN_171 GBV_ILN_187 GBV_ILN_213 GBV_ILN_224 GBV_ILN_230 GBV_ILN_250 GBV_ILN_281 GBV_ILN_285 GBV_ILN_293 GBV_ILN_370 GBV_ILN_602 GBV_ILN_636 GBV_ILN_702 GBV_ILN_2001 GBV_ILN_2003 GBV_ILN_2004 GBV_ILN_2005 GBV_ILN_2006 GBV_ILN_2007 GBV_ILN_2008 GBV_ILN_2009 GBV_ILN_2010 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2015 GBV_ILN_2020 GBV_ILN_2021 GBV_ILN_2025 GBV_ILN_2026 GBV_ILN_2027 GBV_ILN_2031 GBV_ILN_2034 GBV_ILN_2037 GBV_ILN_2038 GBV_ILN_2039 GBV_ILN_2044 GBV_ILN_2048 GBV_ILN_2049 GBV_ILN_2050 GBV_ILN_2055 GBV_ILN_2056 GBV_ILN_2057 GBV_ILN_2059 GBV_ILN_2061 GBV_ILN_2064 GBV_ILN_2065 GBV_ILN_2068 GBV_ILN_2088 GBV_ILN_2093 GBV_ILN_2106 GBV_ILN_2107 GBV_ILN_2108 GBV_ILN_2110 GBV_ILN_2111 GBV_ILN_2112 GBV_ILN_2113 GBV_ILN_2118 GBV_ILN_2122 GBV_ILN_2129 GBV_ILN_2143 GBV_ILN_2144 GBV_ILN_2147 GBV_ILN_2148 GBV_ILN_2152 GBV_ILN_2153 GBV_ILN_2190 GBV_ILN_2232 GBV_ILN_2336 GBV_ILN_2446 GBV_ILN_2470 GBV_ILN_2472 GBV_ILN_2507 GBV_ILN_2522 GBV_ILN_4012 GBV_ILN_4035 GBV_ILN_4037 GBV_ILN_4046 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4242 GBV_ILN_4246 GBV_ILN_4249 GBV_ILN_4251 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4326 GBV_ILN_4328 GBV_ILN_4333 GBV_ILN_4334 GBV_ILN_4335 GBV_ILN_4336 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4393 GBV_ILN_4700 GBV_ILN_2403 GBV_ILN_2403 ISIL_DE-LFER AR 36 2022 8 2108-2120 13 2013 01 DE-16-250 4400302644 00 --%%-- --%%-- --%%-- --%%-- l01 31-10-23 2403 01 DE-LFER 4409565559 00 --%%-- --%%-- n --%%-- l01 16-11-23 2403 01 DE-LFER https://doi.org/10.1038/s41375-022-01615-z 2403 01 DE-LFER http://www.nature.com/articles/s41375-022-01615-z 2013 01 DE-16-250 00 s hd2022 2013 01 DE-16-250 01 s (DE-627)1410508463 wissenschaftlicher Artikel (Zeitschrift) 2013 01 DE-16-250 02 s per_21 2013 01 DE-16-250 03 s s_13 2013 01 DE-16-250 04 p (DE-627)1446321282 Reiter, Andreas 2013 01 DE-16-250 04 k (DE-627)1416468528 III. Medizinische Klinik 2013 01 DE-16-250 04 s (DE-627)1410501914 Verfasser 2013 01 DE-16-250 04 s pos_1 2013 01 DE-16-250 05 p (DE-627)1668484099 Lübke, Johannes 2013 01 DE-16-250 05 k (DE-627)1416468528 III. Medizinische Klinik 2013 01 DE-16-250 05 s (DE-627)1410501914 Verfasser 2013 01 DE-16-250 05 s pos_5 2013 01 DE-16-250 06 p (DE-627)1477558896 Schwaab, Juliana 2013 01 DE-16-250 06 k (DE-627)1416468528 III. Medizinische Klinik 2013 01 DE-16-250 06 s (DE-627)1410501914 Verfasser 2013 01 DE-16-250 06 s pos_12 |
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10.1038/s41375-022-01615-z doi (DE-627)1868839656 (DE-599)KXP1868839656 (OCoLC)1425216584 DE-627 ger DE-627 rda eng Reiter, Andreas 1964- verfasserin (DE-588)172629551 (DE-627)697563766 (DE-576)133488594 aut Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis myelodysplastic syndrome Andreas Reiter, Jason Gotlib, Iván Álvarez-Twose, Deepti H. Radia, Johannes Lübke, Priyanka J. Bobbili, Aolin Wang, Chelsea Norregaard, Saša Dimitrijevic, Erin Sullivan, Melinda Louie-Gao, Juliana Schwaab, Ilene A. Galinsky, Cecelia Perkins, Wolfgang R. Sperr, Priya Sriskandarajah, Andi Chin, Selvam R. Sendhil, Mei Sheng Duh, Peter Valent and Daniel J. DeAngelo 05 July 2022 13 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Gesehen am 31.10.2023 Advanced systemic mastocytosis (AdvSM) is a rare myeloid neoplasm associated with poor overall survival (OS). This study (NCT04695431) compared clinical outcomes between patients with AdvSM treated with avapritinib in the Phase 1 EXPLORER (NCT0256198) and Phase 2 PATHFINDER (NCT03580655) trials (N = 176) and patients treated with best available therapy (BAT; N = 141). A multi-center, observational, retrospective chart review study was conducted at six study sites (four European, two American) to collect data from patients with AdvSM who received BAT; these data were pooled with data from EXPLORER and PATHFINDER. Comparisons between outcomes of OS, duration of treatment (DOT), and maximum reduction in serum tryptase were conducted between the treatment cohorts, with adjustment for key covariates. The results indicated that the avapritinib cohort had significantly better survival (adjusted hazard ratio (HR) (95% confidence interval (CI)): 0.48 (0.29, 0.79); p = 0.004) and significantly longer DOT (HR: 0.36 (0.26, 0.51); p < 0.001) compared to the BAT cohort. Additionally, the mean difference in percentage maximum reduction in serum tryptase levels was 60.3% greater in the avapritinib cohort (95% CI: −72.8, −47.9; p < 0.001). With no randomized controlled trials comparing avapritinib to BAT, these data offer crucial insights into the improved efficacy of avapritinib for the treatment of AdvSM. Cancer therapy Targeted therapies Gotlib, Jason verfasserin aut Álvarez-Twose, Iván verfasserin aut Radia, Deepti H. verfasserin aut Lübke, Johannes 1996- verfasserin (DE-588)1189769867 (DE-627)1668483645 aut Bobbili, Priyanka J. verfasserin aut Wang, Aolin verfasserin aut Norregaard, Chelsea verfasserin aut Dimitrijevic, Saša verfasserin aut Sullivan, Erin verfasserin aut Louie-Gao, Melinda verfasserin aut Schwaab, Juliana 1982- verfasserin (DE-588)1051912687 (DE-627)787019399 (DE-576)407558764 aut Galinsky, Ilene A. verfasserin aut Perkins, Cecelia verfasserin aut Sperr, Wolfgang R. verfasserin aut Sriskandarajah, Priya verfasserin aut Chin, Andi verfasserin aut Sendhil, Selvam R. verfasserin aut Duh, Mei Sheng verfasserin aut Valent, Peter verfasserin aut DeAngelo, Daniel J. verfasserin aut Enthalten in Leukemia London : Springer Nature, 1997 36(2022), 8, Seite 2108-2120 Online-Ressource (DE-627)32046699X (DE-600)2008023-2 (DE-576)094139733 1476-5551 nnns volume:36 year:2022 number:8 pages:2108-2120 extent:13 https://doi.org/10.1038/s41375-022-01615-z Verlag Resolving-System kostenfrei Volltext http://www.nature.com/articles/s41375-022-01615-z Verlag kostenfrei Volltext GBV_USEFLAG_U GBV_ILN_2013 ISIL_DE-16-250 SYSFLAG_1 GBV_KXP GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_32 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_70 GBV_ILN_73 GBV_ILN_74 GBV_ILN_90 GBV_ILN_95 GBV_ILN_100 GBV_ILN_101 GBV_ILN_105 GBV_ILN_110 GBV_ILN_138 GBV_ILN_150 GBV_ILN_151 GBV_ILN_152 GBV_ILN_161 GBV_ILN_170 GBV_ILN_171 GBV_ILN_187 GBV_ILN_213 GBV_ILN_224 GBV_ILN_230 GBV_ILN_250 GBV_ILN_281 GBV_ILN_285 GBV_ILN_293 GBV_ILN_370 GBV_ILN_602 GBV_ILN_636 GBV_ILN_702 GBV_ILN_2001 GBV_ILN_2003 GBV_ILN_2004 GBV_ILN_2005 GBV_ILN_2006 GBV_ILN_2007 GBV_ILN_2008 GBV_ILN_2009 GBV_ILN_2010 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2015 GBV_ILN_2020 GBV_ILN_2021 GBV_ILN_2025 GBV_ILN_2026 GBV_ILN_2027 GBV_ILN_2031 GBV_ILN_2034 GBV_ILN_2037 GBV_ILN_2038 GBV_ILN_2039 GBV_ILN_2044 GBV_ILN_2048 GBV_ILN_2049 GBV_ILN_2050 GBV_ILN_2055 GBV_ILN_2056 GBV_ILN_2057 GBV_ILN_2059 GBV_ILN_2061 GBV_ILN_2064 GBV_ILN_2065 GBV_ILN_2068 GBV_ILN_2088 GBV_ILN_2093 GBV_ILN_2106 GBV_ILN_2107 GBV_ILN_2108 GBV_ILN_2110 GBV_ILN_2111 GBV_ILN_2112 GBV_ILN_2113 GBV_ILN_2118 GBV_ILN_2122 GBV_ILN_2129 GBV_ILN_2143 GBV_ILN_2144 GBV_ILN_2147 GBV_ILN_2148 GBV_ILN_2152 GBV_ILN_2153 GBV_ILN_2190 GBV_ILN_2232 GBV_ILN_2336 GBV_ILN_2446 GBV_ILN_2470 GBV_ILN_2472 GBV_ILN_2507 GBV_ILN_2522 GBV_ILN_4012 GBV_ILN_4035 GBV_ILN_4037 GBV_ILN_4046 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4242 GBV_ILN_4246 GBV_ILN_4249 GBV_ILN_4251 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4326 GBV_ILN_4328 GBV_ILN_4333 GBV_ILN_4334 GBV_ILN_4335 GBV_ILN_4336 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4393 GBV_ILN_4700 GBV_ILN_2403 GBV_ILN_2403 ISIL_DE-LFER AR 36 2022 8 2108-2120 13 2013 01 DE-16-250 4400302644 00 --%%-- --%%-- --%%-- --%%-- l01 31-10-23 2403 01 DE-LFER 4409565559 00 --%%-- --%%-- n --%%-- l01 16-11-23 2403 01 DE-LFER https://doi.org/10.1038/s41375-022-01615-z 2403 01 DE-LFER http://www.nature.com/articles/s41375-022-01615-z 2013 01 DE-16-250 00 s hd2022 2013 01 DE-16-250 01 s (DE-627)1410508463 wissenschaftlicher Artikel (Zeitschrift) 2013 01 DE-16-250 02 s per_21 2013 01 DE-16-250 03 s s_13 2013 01 DE-16-250 04 p (DE-627)1446321282 Reiter, Andreas 2013 01 DE-16-250 04 k (DE-627)1416468528 III. 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Reiter, Andreas 1964- misc Cancer therapy misc Targeted therapies 2013 hd2022 2013 wissenschaftlicher Artikel (Zeitschrift) 2013 per_21 2013 s_13 2013 Reiter, Andreas 2013 III. Medizinische Klinik 2013 Verfasser 2013 pos_1 2013 Lübke, Johannes 2013 pos_5 2013 Schwaab, Juliana 2013 pos_12 Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis myelodysplastic syndrome |
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2013 01 DE-16-250 00 s hd2022 2013 01 DE-16-250 01 s (DE-627)1410508463 wissenschaftlicher Artikel (Zeitschrift) 2013 01 DE-16-250 02 s per_21 2013 01 DE-16-250 03 s s_13 2013 01 DE-16-250 04 p (DE-627)1446321282 Reiter, Andreas 2013 01 DE-16-250 04 k (DE-627)1416468528 III. Medizinische Klinik 2013 01 DE-16-250 04 s (DE-627)1410501914 Verfasser 2013 01 DE-16-250 04 s pos_1 2013 01 DE-16-250 05 p (DE-627)1668484099 Lübke, Johannes 2013 01 DE-16-250 05 k (DE-627)1416468528 III. Medizinische Klinik 2013 01 DE-16-250 05 s (DE-627)1410501914 Verfasser 2013 01 DE-16-250 05 s pos_5 2013 01 DE-16-250 06 p (DE-627)1477558896 Schwaab, Juliana 2013 01 DE-16-250 06 k (DE-627)1416468528 III. Medizinische Klinik 2013 01 DE-16-250 06 s (DE-627)1410501914 Verfasser 2013 01 DE-16-250 06 s pos_12 Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis myelodysplastic syndrome Andreas Reiter, Jason Gotlib, Iván Álvarez-Twose, Deepti H. Radia, Johannes Lübke, Priyanka J. Bobbili, Aolin Wang, Chelsea Norregaard, Saša Dimitrijevic, Erin Sullivan, Melinda Louie-Gao, Juliana Schwaab, Ilene A. Galinsky, Cecelia Perkins, Wolfgang R. Sperr, Priya Sriskandarajah, Andi Chin, Selvam R. Sendhil, Mei Sheng Duh, Peter Valent and Daniel J. DeAngelo Cancer therapy Targeted therapies |
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Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis myelodysplastic syndrome |
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Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis myelodysplastic syndrome Andreas Reiter, Jason Gotlib, Iván Álvarez-Twose, Deepti H. Radia, Johannes Lübke, Priyanka J. Bobbili, Aolin Wang, Chelsea Norregaard, Saša Dimitrijevic, Erin Sullivan, Melinda Louie-Gao, Juliana Schwaab, Ilene A. Galinsky, Cecelia Perkins, Wolfgang R. Sperr, Priya Sriskandarajah, Andi Chin, Selvam R. Sendhil, Mei Sheng Duh, Peter Valent and Daniel J. DeAngelo |
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Reiter, Andreas Gotlib, Jason Álvarez-Twose, Iván Radia, Deepti H. Lübke, Johannes Bobbili, Priyanka J. Wang, Aolin Norregaard, Chelsea Dimitrijevic, Saša Sullivan, Erin Louie-Gao, Melinda Schwaab, Juliana Galinsky, Ilene A. Perkins, Cecelia Sperr, Wolfgang R. Sriskandarajah, Priya Chin, Andi Sendhil, Selvam R. Duh, Mei Sheng Valent, Peter DeAngelo, Daniel J. |
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efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosismyelodysplastic syndrome |
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Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis myelodysplastic syndrome |
abstract |
Advanced systemic mastocytosis (AdvSM) is a rare myeloid neoplasm associated with poor overall survival (OS). This study (NCT04695431) compared clinical outcomes between patients with AdvSM treated with avapritinib in the Phase 1 EXPLORER (NCT0256198) and Phase 2 PATHFINDER (NCT03580655) trials (N = 176) and patients treated with best available therapy (BAT; N = 141). A multi-center, observational, retrospective chart review study was conducted at six study sites (four European, two American) to collect data from patients with AdvSM who received BAT; these data were pooled with data from EXPLORER and PATHFINDER. Comparisons between outcomes of OS, duration of treatment (DOT), and maximum reduction in serum tryptase were conducted between the treatment cohorts, with adjustment for key covariates. The results indicated that the avapritinib cohort had significantly better survival (adjusted hazard ratio (HR) (95% confidence interval (CI)): 0.48 (0.29, 0.79); p = 0.004) and significantly longer DOT (HR: 0.36 (0.26, 0.51); p < 0.001) compared to the BAT cohort. Additionally, the mean difference in percentage maximum reduction in serum tryptase levels was 60.3% greater in the avapritinib cohort (95% CI: −72.8, −47.9; p < 0.001). With no randomized controlled trials comparing avapritinib to BAT, these data offer crucial insights into the improved efficacy of avapritinib for the treatment of AdvSM. Gesehen am 31.10.2023 |
abstractGer |
Advanced systemic mastocytosis (AdvSM) is a rare myeloid neoplasm associated with poor overall survival (OS). This study (NCT04695431) compared clinical outcomes between patients with AdvSM treated with avapritinib in the Phase 1 EXPLORER (NCT0256198) and Phase 2 PATHFINDER (NCT03580655) trials (N = 176) and patients treated with best available therapy (BAT; N = 141). A multi-center, observational, retrospective chart review study was conducted at six study sites (four European, two American) to collect data from patients with AdvSM who received BAT; these data were pooled with data from EXPLORER and PATHFINDER. Comparisons between outcomes of OS, duration of treatment (DOT), and maximum reduction in serum tryptase were conducted between the treatment cohorts, with adjustment for key covariates. The results indicated that the avapritinib cohort had significantly better survival (adjusted hazard ratio (HR) (95% confidence interval (CI)): 0.48 (0.29, 0.79); p = 0.004) and significantly longer DOT (HR: 0.36 (0.26, 0.51); p < 0.001) compared to the BAT cohort. Additionally, the mean difference in percentage maximum reduction in serum tryptase levels was 60.3% greater in the avapritinib cohort (95% CI: −72.8, −47.9; p < 0.001). With no randomized controlled trials comparing avapritinib to BAT, these data offer crucial insights into the improved efficacy of avapritinib for the treatment of AdvSM. Gesehen am 31.10.2023 |
abstract_unstemmed |
Advanced systemic mastocytosis (AdvSM) is a rare myeloid neoplasm associated with poor overall survival (OS). This study (NCT04695431) compared clinical outcomes between patients with AdvSM treated with avapritinib in the Phase 1 EXPLORER (NCT0256198) and Phase 2 PATHFINDER (NCT03580655) trials (N = 176) and patients treated with best available therapy (BAT; N = 141). A multi-center, observational, retrospective chart review study was conducted at six study sites (four European, two American) to collect data from patients with AdvSM who received BAT; these data were pooled with data from EXPLORER and PATHFINDER. Comparisons between outcomes of OS, duration of treatment (DOT), and maximum reduction in serum tryptase were conducted between the treatment cohorts, with adjustment for key covariates. The results indicated that the avapritinib cohort had significantly better survival (adjusted hazard ratio (HR) (95% confidence interval (CI)): 0.48 (0.29, 0.79); p = 0.004) and significantly longer DOT (HR: 0.36 (0.26, 0.51); p < 0.001) compared to the BAT cohort. Additionally, the mean difference in percentage maximum reduction in serum tryptase levels was 60.3% greater in the avapritinib cohort (95% CI: −72.8, −47.9; p < 0.001). With no randomized controlled trials comparing avapritinib to BAT, these data offer crucial insights into the improved efficacy of avapritinib for the treatment of AdvSM. Gesehen am 31.10.2023 |
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Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis |
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score |
7.401039 |