RESUMEN
Introduction: CAR T-cell therapy has emerged as a promising new immuno-oncology treatment that engages the patient's immune system to fight certain hematological malignancies, including diffuse large B-cell lymphoma (DLBCL). In the European Union (EU), CAR T-cell therapies have been approved for relapsed/refractory (R/R) DLBCL patients since 2018, but patient access is often still limited or delayed. This paper is aimed at discussing challenges to access and possible solutions in the largest four EU countries. Methods: The analysis relied on literature review, market data collection, since homogeneous data coming from registries were not available, and discussion with experts coming from all four countries. Results: We calculated that in 2020, between 58% and 83% of R/R DLBCL patients (EMA approved label population) or between 29% and 71% of the estimated medically eligible R/R DLBCL patients, were not treated with a licensed CAR T-cell therapy. Common challenges along the patient journey that may result in limited access or delays to CAR T-cell therapy were identified. These include timely identification and referral of eligible patients, pre-treatment funding approval by authorities and payers, and resource needs at CAR T-cell centers. Discussion: These challenges, existing best practices and recommended focus areas for health systems are discussed here, with the aim to inform necessary actions for overcoming patient access challenges for current CAR T-cell therapies as well as for future cell and gene therapies.
RESUMEN
Patients undergoing allogeneic hematopoietic stem cell transplantation have a high morbidity and mortality, especially after admission to intensive care unit (ICU) during peri-transplant period. The objective of this study was to identify new clinical and biological parameters and validate prognostic scores associated with ICU, short-and long-term survival. Significant differences between ICU survivors and ICU non-survivors for the clinical parameters invasive mechanical ventilation, urine output, heart rate, mean arterial pressure, and amount of vasopressors have been measured. Among prognostic scores (SOFA, SAPSII, PICAT, APACHE II, APACHE IV) assessing severity of disease and predicting outcome of critically ill patients on ICU, the APACHE II score has shown most significant difference (p = 0.002) and the highest discriminative power (area under the ROC curve (AUC) 0.74). An elevated level of lactate at day of admission was associated with poor survival on ICU and the most significant independent parameter (p < 0.001). In our cohort kidney damage with low urine output has a highly relevant impact on ICU, short- and long-term overall survival. The APACHE II score was superior predicting ICU mortality compared to all other tested prognostic scores for patients on ICU during peri-transplant period.
