LOW-DOSE RITUXIMAB AS PRE-EMPTIVE THERAPY IN MULTI-RELAPSING AND PRIMARY REFRACTORY PATIENTS WITH ACQUIRED IDIOPATHIC THROMBOTIC THROMBOCYTOPENIC PURPURA: A MONOCENTRIC RETROSPECTIVE STUDY.
(Abstract release date: 05/19/16)
EHA Library. Fianchi L. 06/09/16; 132977; E1428
Dr. Luana Fianchi
Contributions
Contributions
Abstract
Abstract: E1428
Type: Eposter Presentation
Background
Acquired idiopathic thrombotic thrombocytopenic purpura (TTP) is a life-threatening microangiopathic disorder for which therapeutic plasma exchange (TPE) and steroids represent the primary standard of care. However, a significant proportion of patients still experiences refractory disease, with exacerbations shortly after a perceived remission, or relapse.
Aims
The aim of this retrospective study was to evaluate the long-term efficacy of rituximab in preventing early death in refractory/exacerbated TTP and avoid relapse in “relapsing” patients.
Methods
We collected retrospectively clinical data on all patients with acquired TTP, admitted to our Hematology Department between January 2005 and December 2015. Forty-eight patients [37 female and 11 male; median age: 42.5 years (range 17-80)] were registered and treated for TTP in this period.
Results
Thirty-six patients were included at their first episode of TTP, while 12 patients had a history of multiple relapses [median 2 (range 2-4)], in the previous 10 years (1995-2004). Nine of 48 patients (19%) were diagnosed with TTP during pregnancy. Median platelet and hemoglobin values at diagnosis were 13x109/l (range 5-49) and 7,9 g/dl (4,2-10.1),respectively. ADAMTS-13 concentration was measured at diagnosis or at relapse in all patients and was <10% in all cases [median 0% (range 0-6)]. Anti-ADAMTS13 autoantibodies were present in all 23 patients tested.All 48 patients received TPE associated to 1mg/kg methyl-prednisolone as frontline therapy. After a median of 10 TPE (range 8-18), 32 patients (67%) (20/36 naïve, 12/12 at relapse) achieved disease remission [normal platelet counts, increase of ADAMTS-13 activity [median 78% ( range 50-215)] and disappearance of inhibitors), while 16 (33%) resulted refractory or experienced exacerbation.Rituximab (RTX), at the dose of 375 mg/m2 weekly for 4 weeks, was then added to TPE in 27 patients (56%), including 15/36 patients at their first TTP episode, who resulted refractory to TPE or showed exacerbation, and in the 12 relapsing patients, to prevent subsequent relapse. One patient with TTP exacerbation died early, prior to RTX start.Complete response to RTX was documented in 12 of 15 refractory patients, and in all 12 relapsing patients. Three patients died early, before the second RTX dose, resulting into 8% overall 30-day mortality rate (4 patients).Mainteinance treatment with low dose RTX (100 mg every three months for 2 years) was then administered to 23 patients (12 relapsing and 11 refractory), while 1 patient refractory to TPE refused maintenance. At a median follow up of 34 months (range 1-87) from the first dose of maintenance none of the patients relapsed, and we did not observe any infectious complications. This resulted into a significant reduction in the incidence of relapses after RTX treatment (HR 4.26, 95% CI 1.511-12.05, p 0.0089)ADAMTS-13 activity was stable at >50% and inhibitors were not detected during follow-up visits, performed every three months in all 23 patients.
Conclusion
Our results suggest that pre-emptive treatment with low dose rituximab, is safe and effective to maintain remission also in patients with primary refractory TTP or with a history of multiple relapse.
Session topic: E-poster
Keyword(s): Relapse, Rituximab, Thrombotic thrombocytopenic purpura (TTP)
Type: Eposter Presentation
Background
Acquired idiopathic thrombotic thrombocytopenic purpura (TTP) is a life-threatening microangiopathic disorder for which therapeutic plasma exchange (TPE) and steroids represent the primary standard of care. However, a significant proportion of patients still experiences refractory disease, with exacerbations shortly after a perceived remission, or relapse.
Aims
The aim of this retrospective study was to evaluate the long-term efficacy of rituximab in preventing early death in refractory/exacerbated TTP and avoid relapse in “relapsing” patients.
Methods
We collected retrospectively clinical data on all patients with acquired TTP, admitted to our Hematology Department between January 2005 and December 2015. Forty-eight patients [37 female and 11 male; median age: 42.5 years (range 17-80)] were registered and treated for TTP in this period.
Results
Thirty-six patients were included at their first episode of TTP, while 12 patients had a history of multiple relapses [median 2 (range 2-4)], in the previous 10 years (1995-2004). Nine of 48 patients (19%) were diagnosed with TTP during pregnancy. Median platelet and hemoglobin values at diagnosis were 13x109/l (range 5-49) and 7,9 g/dl (4,2-10.1),respectively. ADAMTS-13 concentration was measured at diagnosis or at relapse in all patients and was <10% in all cases [median 0% (range 0-6)]. Anti-ADAMTS13 autoantibodies were present in all 23 patients tested.All 48 patients received TPE associated to 1mg/kg methyl-prednisolone as frontline therapy. After a median of 10 TPE (range 8-18), 32 patients (67%) (20/36 naïve, 12/12 at relapse) achieved disease remission [normal platelet counts, increase of ADAMTS-13 activity [median 78% ( range 50-215)] and disappearance of inhibitors), while 16 (33%) resulted refractory or experienced exacerbation.Rituximab (RTX), at the dose of 375 mg/m2 weekly for 4 weeks, was then added to TPE in 27 patients (56%), including 15/36 patients at their first TTP episode, who resulted refractory to TPE or showed exacerbation, and in the 12 relapsing patients, to prevent subsequent relapse. One patient with TTP exacerbation died early, prior to RTX start.Complete response to RTX was documented in 12 of 15 refractory patients, and in all 12 relapsing patients. Three patients died early, before the second RTX dose, resulting into 8% overall 30-day mortality rate (4 patients).Mainteinance treatment with low dose RTX (100 mg every three months for 2 years) was then administered to 23 patients (12 relapsing and 11 refractory), while 1 patient refractory to TPE refused maintenance. At a median follow up of 34 months (range 1-87) from the first dose of maintenance none of the patients relapsed, and we did not observe any infectious complications. This resulted into a significant reduction in the incidence of relapses after RTX treatment (HR 4.26, 95% CI 1.511-12.05, p 0.0089)ADAMTS-13 activity was stable at >50% and inhibitors were not detected during follow-up visits, performed every three months in all 23 patients.
Conclusion
Our results suggest that pre-emptive treatment with low dose rituximab, is safe and effective to maintain remission also in patients with primary refractory TTP or with a history of multiple relapse.
Session topic: E-poster
Keyword(s): Relapse, Rituximab, Thrombotic thrombocytopenic purpura (TTP)
Abstract: E1428
Type: Eposter Presentation
Background
Acquired idiopathic thrombotic thrombocytopenic purpura (TTP) is a life-threatening microangiopathic disorder for which therapeutic plasma exchange (TPE) and steroids represent the primary standard of care. However, a significant proportion of patients still experiences refractory disease, with exacerbations shortly after a perceived remission, or relapse.
Aims
The aim of this retrospective study was to evaluate the long-term efficacy of rituximab in preventing early death in refractory/exacerbated TTP and avoid relapse in “relapsing” patients.
Methods
We collected retrospectively clinical data on all patients with acquired TTP, admitted to our Hematology Department between January 2005 and December 2015. Forty-eight patients [37 female and 11 male; median age: 42.5 years (range 17-80)] were registered and treated for TTP in this period.
Results
Thirty-six patients were included at their first episode of TTP, while 12 patients had a history of multiple relapses [median 2 (range 2-4)], in the previous 10 years (1995-2004). Nine of 48 patients (19%) were diagnosed with TTP during pregnancy. Median platelet and hemoglobin values at diagnosis were 13x109/l (range 5-49) and 7,9 g/dl (4,2-10.1),respectively. ADAMTS-13 concentration was measured at diagnosis or at relapse in all patients and was <10% in all cases [median 0% (range 0-6)]. Anti-ADAMTS13 autoantibodies were present in all 23 patients tested.All 48 patients received TPE associated to 1mg/kg methyl-prednisolone as frontline therapy. After a median of 10 TPE (range 8-18), 32 patients (67%) (20/36 naïve, 12/12 at relapse) achieved disease remission [normal platelet counts, increase of ADAMTS-13 activity [median 78% ( range 50-215)] and disappearance of inhibitors), while 16 (33%) resulted refractory or experienced exacerbation.Rituximab (RTX), at the dose of 375 mg/m2 weekly for 4 weeks, was then added to TPE in 27 patients (56%), including 15/36 patients at their first TTP episode, who resulted refractory to TPE or showed exacerbation, and in the 12 relapsing patients, to prevent subsequent relapse. One patient with TTP exacerbation died early, prior to RTX start.Complete response to RTX was documented in 12 of 15 refractory patients, and in all 12 relapsing patients. Three patients died early, before the second RTX dose, resulting into 8% overall 30-day mortality rate (4 patients).Mainteinance treatment with low dose RTX (100 mg every three months for 2 years) was then administered to 23 patients (12 relapsing and 11 refractory), while 1 patient refractory to TPE refused maintenance. At a median follow up of 34 months (range 1-87) from the first dose of maintenance none of the patients relapsed, and we did not observe any infectious complications. This resulted into a significant reduction in the incidence of relapses after RTX treatment (HR 4.26, 95% CI 1.511-12.05, p 0.0089)ADAMTS-13 activity was stable at >50% and inhibitors were not detected during follow-up visits, performed every three months in all 23 patients.
Conclusion
Our results suggest that pre-emptive treatment with low dose rituximab, is safe and effective to maintain remission also in patients with primary refractory TTP or with a history of multiple relapse.
Session topic: E-poster
Keyword(s): Relapse, Rituximab, Thrombotic thrombocytopenic purpura (TTP)
Type: Eposter Presentation
Background
Acquired idiopathic thrombotic thrombocytopenic purpura (TTP) is a life-threatening microangiopathic disorder for which therapeutic plasma exchange (TPE) and steroids represent the primary standard of care. However, a significant proportion of patients still experiences refractory disease, with exacerbations shortly after a perceived remission, or relapse.
Aims
The aim of this retrospective study was to evaluate the long-term efficacy of rituximab in preventing early death in refractory/exacerbated TTP and avoid relapse in “relapsing” patients.
Methods
We collected retrospectively clinical data on all patients with acquired TTP, admitted to our Hematology Department between January 2005 and December 2015. Forty-eight patients [37 female and 11 male; median age: 42.5 years (range 17-80)] were registered and treated for TTP in this period.
Results
Thirty-six patients were included at their first episode of TTP, while 12 patients had a history of multiple relapses [median 2 (range 2-4)], in the previous 10 years (1995-2004). Nine of 48 patients (19%) were diagnosed with TTP during pregnancy. Median platelet and hemoglobin values at diagnosis were 13x109/l (range 5-49) and 7,9 g/dl (4,2-10.1),respectively. ADAMTS-13 concentration was measured at diagnosis or at relapse in all patients and was <10% in all cases [median 0% (range 0-6)]. Anti-ADAMTS13 autoantibodies were present in all 23 patients tested.All 48 patients received TPE associated to 1mg/kg methyl-prednisolone as frontline therapy. After a median of 10 TPE (range 8-18), 32 patients (67%) (20/36 naïve, 12/12 at relapse) achieved disease remission [normal platelet counts, increase of ADAMTS-13 activity [median 78% ( range 50-215)] and disappearance of inhibitors), while 16 (33%) resulted refractory or experienced exacerbation.Rituximab (RTX), at the dose of 375 mg/m2 weekly for 4 weeks, was then added to TPE in 27 patients (56%), including 15/36 patients at their first TTP episode, who resulted refractory to TPE or showed exacerbation, and in the 12 relapsing patients, to prevent subsequent relapse. One patient with TTP exacerbation died early, prior to RTX start.Complete response to RTX was documented in 12 of 15 refractory patients, and in all 12 relapsing patients. Three patients died early, before the second RTX dose, resulting into 8% overall 30-day mortality rate (4 patients).Mainteinance treatment with low dose RTX (100 mg every three months for 2 years) was then administered to 23 patients (12 relapsing and 11 refractory), while 1 patient refractory to TPE refused maintenance. At a median follow up of 34 months (range 1-87) from the first dose of maintenance none of the patients relapsed, and we did not observe any infectious complications. This resulted into a significant reduction in the incidence of relapses after RTX treatment (HR 4.26, 95% CI 1.511-12.05, p 0.0089)ADAMTS-13 activity was stable at >50% and inhibitors were not detected during follow-up visits, performed every three months in all 23 patients.
Conclusion
Our results suggest that pre-emptive treatment with low dose rituximab, is safe and effective to maintain remission also in patients with primary refractory TTP or with a history of multiple relapse.
Session topic: E-poster
Keyword(s): Relapse, Rituximab, Thrombotic thrombocytopenic purpura (TTP)
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