Background and Objectives: The treatment of chronic lymphocytic leukemia (CLL) has acquired new targeted therapies. In clinical trials, ibrutinib improved outcomes safely. Real-world data called for a reappraisal of ibrutinib strategies. We report on a single center’s experience with ibrutinib monotherapy, aiming to explore the outcomes, tolerability, and prognosis of CLL patients in routine clinical practice. Materials and Methods: Data were collected from all CLL patients treated with ibrutinib at Fundeni Clinical Institute, Bucharest, Romania, between January 2016 and June 2021. Results: A total of one hundred twenty-three CLL adult patients were treated with ibrutinib. Of the patients, 87% had relapsed/refractory CLL. The median age at ibrutinib initiation was 65 years; 44.7% of patients were staged Rai III/IV. At 32-month median follow-up, the median progression-free survival (PFS) was 50 months, the overall survival (OS) was not reached, and the overall response rate (ORR) was 86.2%. The age or number of previous therapies did not impact outcomes or tolerability. An Eastern Cooperative Oncology Group performance status (ECOG PS) score ≥ 2 and shorter time from initiation of last therapy (TILT) before ibrutinib predicted inferior PFS. Baseline characteristics had no impact on the OS except for TILT in R/R CLL patients. Drug-related adverse events (AEs) of any grade and grade ≥ 3 AEs were reported in 82.1% and 30.9% of the patients, respectively. Infections were the most common AEs (29.3%). Drug discontinuation was permanent in 43.9% of patients, mainly due to disease progression (17.1%) and toxicity (8.9%). Patients with a Cumulative Illness Rating Scale (CIRS) score ≥ 6 had a higher risk for toxicity-related discontinuation. An ECOG PS ≥ 2 predicted an increased rate of permanent discontinuation and grade ≥ 3 AEs. Conclusions: The outcomes of this study align with the results from ibrutinib clinical trials. Our study demonstrated that poor patient fitness, early relapse before ibrutinib, and permanent ibrutinib discontinuation are essential outcome determinants. Patient comorbidity burden and fitness were significant predictors for ibrutinib intolerance.
Background: Thrombotic thrombocytopenic purpura (TTP) is a rare and life-threatening thrombotic microangiopathy requiring prompt diagnosis and treatment. This retrospective single-center study analyzed 31 adult patients diagnosed between 2013 and 2024 (PLASMIC score ≥ 5, ADAMTS13 activity < 10%), aiming to characterize their clinical profiles and assess the impact of caplacizumab. Methods: Baseline laboratory parameters (platelet count, LDH, creatinine, hemoglobin, number of plasmapheresis sessions, number of hospitalization days, number of days in intensive care, and days required to recover platelet count) were included in statistical analysis to predict diverse outcomes, such as respiratory distress, infection, major neurological manifestations, gastrointestinal involvement, or refractoriness/exacerbation. Sixteen patients underwent treatment with caplacizumab in addition to plasmapheresis (PEX) and corticosteroids, while the remainder received PEX and corticosteroids alone. Results: Our predictive models proved noteworthy, providing results with ROC values ranging from 0.80 to 0.90 (p < 0.01). Caplacizumab was associated with faster platelet recovery (4 days vs. 7 days), fewer PEX sessions, shorter hospital stays, and a significantly lower incidence of refractoriness or exacerbation (p < 0.05). Inter-group analysis confirmed a significant reduction of overall resource use (p < 0.05). Conclusions: Early caplacizumab use improved outcomes and optimized resource utilization. This real-world study suggests that routinely available laboratory markers at presentation can help predict outcomes and guide early clinical decisions in centers without rapid ADAMTS13 testing.