Observational studyBritish journal of haematology2024
Clinical outcomes in patients in any phase of CML treated with ponatinib in France-Data from the TOPASE observational study.
Observational study in British journal of haematology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04048564 (Observational Study on CML Patients in Any Phase of the Disease Treated With Ponatinib), which is not on this map. Cited by 4 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Observational Study on CML Patients in Any Phase of the Disease Treated With Ponatinib (Iclusig®): A Multicentre, Ambispective Cohort Study
Who cites it
4 citing papers in PubMed.
- Real-world outcomes of ponatinib in heavily pretreated patients with chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia.Annals of hematology · 2026Article
- Article
- Ponatinib for CML patients in routine clinical practice: the PONDEROSA study.Annals of hematology · 2026Observational
- Clinical outcomes in patients in any phase of CML treated with ponatinib in France-Data from the TOPASE observational study.British journal of haematology · 2024Observational
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
23 authors.
Funding
Abstract
The TOPASE study was set up to evaluate the outcomes of chronic myeloid leukaemia [CML] patients treated with ponatinib (PON) in a real-world setting in France. One hundred and twenty CML patients, 105 in chronic phase (CP), 8 in accelerated phase (AP) and 7 in blastic phase (BP) were included. Fifty-one (49%) of the CP-CML patients were in third line of treatment. The trigger for PON initiation in CP-CML was 'poor response' in 67 patients, 'poor tolerance' in 28 patients and 'response enhancement' in seven patients. The median dose at initiation was 30 mg/day [Q1; Q3 = 15; 30] in CP-CML and 45 mg/day [Q1; Q3 = 30; 45] in AP/BP-CML. Of 98 CP-CML evaluable patients, 72 (73.5%) were considered as responders (MMR) at one time point at least once, especially for those in second line of treatment and/or presenting a T315I mutation. Ninety-six of 120 (80%) patients reported at least one adverse event. An arterial occlusive event (AOE) was reported in 11 patients (9.2%). Thus, these real-life data confirm the potency of ponatinib in resistant or intolerant patients with an acceptable safety profile in non-selected patients. NCT number: NCT04048564.
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Registered trials
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