Contributions
Abstract: S141
Type: Oral Presentation
Presentation during EHA23: On Friday, June 15, 2018 from 12:00 - 12:15
Location: Room A9
Background
The long-term safety and efficacy of rituximab (RTX) in adult's immune thrombocytopenia (ITP) are not well known and current data are only based on retrospective studies.
Aims
To assess long-term (5 to 7 years of follow-up) safety and efficacy of RTX in ITP throughout the prospective registry set up in France in 2010 in which 248 adult patients were included (ClinicalTrials.Gov: NCT1101295).
Methods
All consecutive patients ≥ 18 years of age who received RTX for a diagnosis of primary ITP based on international criteria were prospectively included between 2010 and 2012. Patients with secondary ITP or who had received a previous course of RTX were excluded. A prospective and periodic assessment of the safety and the efficacy of RTX were recorded through an electronic case report form. In accordance to international guidelines, complete response (CR) was defined by a platelet count >100x109/L and response (R) by a platelet count between 30-100x109/L with at least a 2-fold increase from baseline. One-year follow-up data have already been published (Khellaf et al., Blood 2014).
Results
Among the 248 patients included in the registry (64% of females, mean age at ITP diagnosis: 51±20 years), 102 (41%) patients had persistent ITP and 146 (59%) chronic ITP at time of first RTX administration, and 10% were splenectomized. The median follow-up duration after the first RTX infusion was 69 [IQR, 55-79] months, with a follow-up ≥60 months for 177 (71%) patients. In terms of efficacy, at last follow-up visit, 77 (31%) patients had a lasting response (70 CR; 7 R). Among the 177 patients with a follow-up ≥60 months, 50 (28%) had a lasting response (46 CR; 4 R). According to National Cancer Institute Common Terminology Criteria, 34 (14%) grade 3 or 4 infections were observed, but only 10 (4%) of them occurred within the 12 months following the last RTX infusion, including 1 pneumocystis pneumonia and 1 aspergillosis sinusitis. No case of progressive multifocal encephalopathy was observed. Malignancies were observed in 24 (10%) patients and occurred at a median age of 71 [62-79] years of age after a median of 48 [39-62] months from RTX (incidence rate of 1.4 [IC95%, 1.1-2.5] for 100 patient-years, similar to the one observed in the French general population). No over-representation of a type of malignancy was found. Moreover, there were 47 adverse effects (AE) related to RTX infusion; 22 (9%) patients developed or exacerbated another auto-immune disease; 21 (8%) patients had cardiovascular complications; and 16 (6%) experienced at least 1 venous thromboembolic event. Overall, 31 (12%) patients died (median age: 80 [IQR, 71-84] years) after a median time of 30 [IQR, 14-54] months after the first RTX infusion, corresponding to a mortality rate of 2.4 [IC95%, 1.7-3.4] for 100 patient-years. Deaths were mainly related to infections (n=6), malignancies (n=5) or bleeding (n=4). Among these AEs, only 24 AE ≥ grade 3 were possibly related to RTX: 10 (4%) infections, 10 (4%) AE related to RTX infusion, 4 (2%) deaths (3 from infectious origin, 1 from unexplained cause). Gammaglobulin levels were not systematically monitored. Among the 142 (57%) patients from whom the data was available, 6 (2%) patients developed a hypogammaglobulinemia <5 g/L during follow-up.
Conclusion
This large nationwide prospective registry shows that a durable response is achieved in almost one third (31%) of adults with persistent or chronic ITP treated by RTX, and that no unexpected long-term complications occur beyond 12 months of follow-up.
Session topic: 33. Platelets disorders
Keyword(s): Immune thrombocytopenia (ITP), Rituximab
Abstract: S141
Type: Oral Presentation
Presentation during EHA23: On Friday, June 15, 2018 from 12:00 - 12:15
Location: Room A9
Background
The long-term safety and efficacy of rituximab (RTX) in adult's immune thrombocytopenia (ITP) are not well known and current data are only based on retrospective studies.
Aims
To assess long-term (5 to 7 years of follow-up) safety and efficacy of RTX in ITP throughout the prospective registry set up in France in 2010 in which 248 adult patients were included (ClinicalTrials.Gov: NCT1101295).
Methods
All consecutive patients ≥ 18 years of age who received RTX for a diagnosis of primary ITP based on international criteria were prospectively included between 2010 and 2012. Patients with secondary ITP or who had received a previous course of RTX were excluded. A prospective and periodic assessment of the safety and the efficacy of RTX were recorded through an electronic case report form. In accordance to international guidelines, complete response (CR) was defined by a platelet count >100x109/L and response (R) by a platelet count between 30-100x109/L with at least a 2-fold increase from baseline. One-year follow-up data have already been published (Khellaf et al., Blood 2014).
Results
Among the 248 patients included in the registry (64% of females, mean age at ITP diagnosis: 51±20 years), 102 (41%) patients had persistent ITP and 146 (59%) chronic ITP at time of first RTX administration, and 10% were splenectomized. The median follow-up duration after the first RTX infusion was 69 [IQR, 55-79] months, with a follow-up ≥60 months for 177 (71%) patients. In terms of efficacy, at last follow-up visit, 77 (31%) patients had a lasting response (70 CR; 7 R). Among the 177 patients with a follow-up ≥60 months, 50 (28%) had a lasting response (46 CR; 4 R). According to National Cancer Institute Common Terminology Criteria, 34 (14%) grade 3 or 4 infections were observed, but only 10 (4%) of them occurred within the 12 months following the last RTX infusion, including 1 pneumocystis pneumonia and 1 aspergillosis sinusitis. No case of progressive multifocal encephalopathy was observed. Malignancies were observed in 24 (10%) patients and occurred at a median age of 71 [62-79] years of age after a median of 48 [39-62] months from RTX (incidence rate of 1.4 [IC95%, 1.1-2.5] for 100 patient-years, similar to the one observed in the French general population). No over-representation of a type of malignancy was found. Moreover, there were 47 adverse effects (AE) related to RTX infusion; 22 (9%) patients developed or exacerbated another auto-immune disease; 21 (8%) patients had cardiovascular complications; and 16 (6%) experienced at least 1 venous thromboembolic event. Overall, 31 (12%) patients died (median age: 80 [IQR, 71-84] years) after a median time of 30 [IQR, 14-54] months after the first RTX infusion, corresponding to a mortality rate of 2.4 [IC95%, 1.7-3.4] for 100 patient-years. Deaths were mainly related to infections (n=6), malignancies (n=5) or bleeding (n=4). Among these AEs, only 24 AE ≥ grade 3 were possibly related to RTX: 10 (4%) infections, 10 (4%) AE related to RTX infusion, 4 (2%) deaths (3 from infectious origin, 1 from unexplained cause). Gammaglobulin levels were not systematically monitored. Among the 142 (57%) patients from whom the data was available, 6 (2%) patients developed a hypogammaglobulinemia <5 g/L during follow-up.
Conclusion
This large nationwide prospective registry shows that a durable response is achieved in almost one third (31%) of adults with persistent or chronic ITP treated by RTX, and that no unexpected long-term complications occur beyond 12 months of follow-up.
Session topic: 33. Platelets disorders
Keyword(s): Immune thrombocytopenia (ITP), Rituximab