Characteristics and Outcomes of Adult Patients in the PETHEMA Registry with Relapsed or Refractory FLT3-ITD Mutation-Positive Acute Myeloid Leukemia
This retrospective study investigated outcomes of 404 patients with relapsed/refractory (R/R) FMS-like tyrosine kinase 3 (FLT3)-internal tandem duplication (ITD) acute myeloid leukemia (AML) enrolled in the PETHEMA registry, pre-approval of tyrosine kinase inhibitors. Most patients (63%) had received first-line intensive therapy with 3 + 7. Subsequently, patients received salvage with intensive therapy (n = 261), non-intensive therapy (n = 63) or supportive care only (n = 80). Active salvage therapy (i.e., intensive or non-intensive therapy) resulted in a complete remission (CR) or CR without hematological recovery (CRi) rate of 42%. More patients achieved a CR/CRi with intensive (48%) compared with non-intensive (19%) salvage therapy (p < 0.001). In the overall population, median overall survival (OS) was 5.5 months; 1- and 5-year OS rates were 25% and 7%. OS was significantly (p < 0.001) prolonged with intensive or non-intensive salvage therapy compared with supportive therapy, and in those achieving CR/CRi versus no responders. Of 280 evaluable patients, 61 (22%) had an allogeneic stem-cell transplant after they had achieved CR/CRi. In conclusion, in this large cohort study, salvage treatment approaches for patients with FLT3-ITD mutated R/R AML were heterogeneous. Median OS was poor with both non-intensive and intensive salvage therapy, with best long-term outcomes obtained in patients who achieved CR/CRi and subsequently underwent allogeneic stem-cell transplant.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2022 |
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| Erschienen: |
2022 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:14 |
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| Enthalten in: |
Cancers - 14(2022), 11 vom: 06. Juni |
| Sprache: |
Englisch |
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| Links: |
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| Themen: |
Acute myeloid leukemia |
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| Anmerkungen: |
Date Revised 08.03.2023 published: Electronic Citation Status PubMed-not-MEDLINE |
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| doi: |
10.3390/cancers14112817 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM342034936 |
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| 100 | 1 | |a Martínez-Cuadrón, David |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Characteristics and Outcomes of Adult Patients in the PETHEMA Registry with Relapsed or Refractory FLT3-ITD Mutation-Positive Acute Myeloid Leukemia |
| 264 | 1 | |c 2022 | |
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| 500 | |a Date Revised 08.03.2023 | ||
| 500 | |a published: Electronic | ||
| 500 | |a Citation Status PubMed-not-MEDLINE | ||
| 520 | |a This retrospective study investigated outcomes of 404 patients with relapsed/refractory (R/R) FMS-like tyrosine kinase 3 (FLT3)-internal tandem duplication (ITD) acute myeloid leukemia (AML) enrolled in the PETHEMA registry, pre-approval of tyrosine kinase inhibitors. Most patients (63%) had received first-line intensive therapy with 3 + 7. Subsequently, patients received salvage with intensive therapy (n = 261), non-intensive therapy (n = 63) or supportive care only (n = 80). Active salvage therapy (i.e., intensive or non-intensive therapy) resulted in a complete remission (CR) or CR without hematological recovery (CRi) rate of 42%. More patients achieved a CR/CRi with intensive (48%) compared with non-intensive (19%) salvage therapy (p < 0.001). In the overall population, median overall survival (OS) was 5.5 months; 1- and 5-year OS rates were 25% and 7%. OS was significantly (p < 0.001) prolonged with intensive or non-intensive salvage therapy compared with supportive therapy, and in those achieving CR/CRi versus no responders. Of 280 evaluable patients, 61 (22%) had an allogeneic stem-cell transplant after they had achieved CR/CRi. In conclusion, in this large cohort study, salvage treatment approaches for patients with FLT3-ITD mutated R/R AML were heterogeneous. Median OS was poor with both non-intensive and intensive salvage therapy, with best long-term outcomes obtained in patients who achieved CR/CRi and subsequently underwent allogeneic stem-cell transplant | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a FLT3-ITD mutation | |
| 650 | 4 | |a acute myeloid leukemia | |
| 650 | 4 | |a real-world outcomes | |
| 650 | 4 | |a relapsed/refractory disease | |
| 650 | 4 | |a salvage therapy | |
| 700 | 1 | |a Serrano, Josefina |e verfasserin |4 aut | |
| 700 | 1 | |a Mariz, José |e verfasserin |4 aut | |
| 700 | 1 | |a Gil, Cristina |e verfasserin |4 aut | |
| 700 | 1 | |a Tormo, Mar |e verfasserin |4 aut | |
| 700 | 1 | |a Martínez-Sánchez, Pilar |e verfasserin |4 aut | |
| 700 | 1 | |a Rodríguez-Arbolí, Eduardo |e verfasserin |4 aut | |
| 700 | 1 | |a García-Boyero, Raimundo |e verfasserin |4 aut | |
| 700 | 1 | |a Rodríguez-Medina, Carlos |e verfasserin |4 aut | |
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| 700 | 1 | |a Polo, Marta |e verfasserin |4 aut | |
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| 700 | 1 | |a Pérez-Encinas, Manuel M |e verfasserin |4 aut | |
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| 700 | 1 | |a López, Juan A |e verfasserin |4 aut | |
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