Publications by authors named "P Cony-Makhoul"

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.

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  • Autologous anti-CD19 CAR T cells are becoming standard treatment for relapsed/refractory large B-cell lymphoma, but serious side effects like cytokine release syndrome (CRS) and neurotoxicity (ICANS) pose risks, often requiring ICU care.
  • In a study involving 925 patients in France, high rates of CRS (84.1%) and ICANS (40.5%) were observed, with significant proportions experiencing severe forms of these conditions.
  • Two prognostic scoring systems (CRS-PSS and ICANS-PSS) were developed to identify patients at higher risk for severe CRS and ICANS based on specific clinical factors, and these scores were validated in other patient groups treated with similar therapies.
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  • In chronic myeloid leukemia, predicting stable treatment-free remission (TFR) after stopping tyrosine kinase inhibitors (TKIs) is difficult, especially using early molecular indicators.
  • A study involving 408 patients compared various predictive tools, including the EUTOS long-term survival score, BCR::ABL1 transcript halving time, and residual disease measurements at months 3 and 6.
  • While months 3 and 6 residual disease showed good predictive performance, particularly month 6, no early indicators effectively forecasted the TKI discontinuation criteria or TFR maintenance, indicating additional factors may be at play.
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  • Histologic transformation of Waldenström macroglobulinemia (HT-WM) usually results in a poor outlook when treated with standard therapies.* -
  • This report presents the first cases of HT-WM being treated with chimeric antigen receptor T cells (CAR-T), which showed promising effectiveness.* -
  • The treatment with CAR-T cells did not cause any unexpected side effects, indicating it might be a safer option for these patients.*
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  • Real-world data (RWD) are crucial for enhancing clinical trial (CT) findings, but challenges like data quality persist; the REALYSA study, launched in 2018, focuses on newly diagnosed lymphoma patients in France.
  • A proof-of-concept analysis of 645 patients with diffuse large B-cell lymphoma (DLBCL) found high data completeness (<4% missing) and revealed good survival rates, with a median follow-up of 9.9 months showing 1-year event-free survival of 77.9% and overall survival of 90.0%.
  • The study also assessed how well REALYSA's patient outcomes matched those from recent phase 3 trials (POLARIX and SENIOR), demonstrating
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