Outcomes 5 years after response to rituximab therapy in children and adults with immune thrombocytopenia

Patel, Vivek; Mahévas, Matthieu; Lee, Soo Y.; Stasi, Roberto; Cunningham‐Rundles, Susanna; Godeau, Bertrand; Kanter, Julie; Neufeld, Ellis J.; Taube, Tillmann; Ramenghi, Ugo; Shenoy, Shalini; Ward, Mary J.; Mihatov, Nino; Patel, Vinay Kumar; Bierling, Philippe; Lesser, Martin; Cooper, Nichola; Bussel, James B.

doi:10.1182/blood-2011-11-393975

Cited by 298 publications

(264 citation statements)

References 47 publications

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“…Less impressive long-term outcome has been on the contrary reported by Patel et al [13] who analyzed data from 17 published studies which included 376 adults showing 5 years response rate only in 21%. This discrepancy could be at least partially explained by ours and Godeau's selection of patients with a lower degree of treatments received before rituximab [19].…”

Section: Discussionmentioning

confidence: 86%

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Long‐term follow‐up analysis after rituximab salvage therapy in adult patients with immune thrombocytopenia

Zaja¹,

Volpetti²,

Chiozzotto³

et al. 2012

American J Hematol

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We report the long-term outcome results of 57 consecutive adult patients with immune thrombocytopenia after being treated with rituximab. According to the different period of therapy, patients received either standard dose (SD) rituximab (i.e., 375 mg/m 2 weekly for 4 weeks) or low dose (LD) rituximab (i.e., 100 mg flat dose weekly for 4 weeks). Overall (OR) and complete response (CR) rates were 60 and 40%, respectively. Patients' median follow-up was 52 months, 82 months in the SD, and 44 months in the LD group; 15 out of 34 responsive patients (44%) relapsed, with median response duration of 24 months (range 3-120). The estimated 4-years event-free survival (EFS, considering events the non response status at month 2 or relapses in responders) was 30%. Patients who received SD vs. LD rituximab had better outcome with regard to short term response (OR 66 vs. 52%, CR 50 vs. 28%), relapse rate (38 vs. 54%), probability to achieve and maintain long-term response (41 vs. 24%) and estimated 4-years EFS (35 vs. 23%). Patients with a longer interval between diagnosis and rituximab therapy had worse EFS [HR 5 1.005; 95%IC: (1.002-1.009), P 5 0.019]. Three patients developed short-term adverse events, two-serum sickness, and one interstitial pneumonia. Four cases of malignancies and two herpes zoster reactivations were registered during long-term follow-up; one patient died for cerebral bleeding. Rituximab SD appears a safe and active agent allowing in nearly 40% of cases to achieve long-term response and splenectomy sparing effect. Am. J. Hematol. 87:886-889, 2012. V

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Section: Discussionmentioning

confidence: 86%

“…Recently, Patel et al [13], analyzing data of 72 adults and 66 children with ITP who had at least an initial response to rituximab, calculated an estimated 5-years response rate of 21 and 30%, respectively.…”

Section: Introductionmentioning

confidence: 99%

Long‐term follow‐up analysis after rituximab salvage therapy in adult patients with immune thrombocytopenia

Zaja¹,

Volpetti²,

Chiozzotto³

et al. 2012

American J Hematol

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“…In France, based on the findings of a single-arm prospective study [4], RTX is now commonly used in persistent or chronic cases of adult ITP prior to splenectomy. Approximately 40-50% of patients treated with RTX at a standard dose achieve a complete response (CR) by 1 year [5] and 20% have a lasting response at 5 years [6]. The mechanisms of action of RTX to treat ITP remain speculative, although the removal of autoreactive B-cells is likely to play an important role.…”

Section: Introductionmentioning

confidence: 99%

Efficacy and safety of rituximab given at 1,000 mg on days 1 and 15 compared to the standard regimen to treat adult immune thrombocytopenia

Mahévas

Ebbo

Audia³

et al. 2013

American J Hematol

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Rituximab (RTX) is used off-label to treat immune thrombocytopenia (ITP) but the regimen now commonly used in rheumatoid arthritis has not been evaluated in ITP. The aim of this large French multicenter retrospective study was to compare the efficacy and safety of two RTX regimens in adult's ITP. The efficacy of two (RTX) regimens: standard therapy of 375 mg/m 2 weekly for 4 weeks vs. a rheumatoid arthritis (RA) regimen of 1,000 mg on days 1 and 15, to treat ITP was compared. We included adults patients with previously primary ITP treated with RTX instead of treated primary ITP. (CR) was defined as a platelet count >100 3 10 9 /L, and a response (R) by a platelet count of >30 3 10 9 /L with a least a doubling of the baseline value. Of the 107 patients included, 61 (57%) received the standard regimen and 46 (43%) the RA regimen. Baseline characteristics and overall response rates at 3 month (M3) and 12 months (M12) were not significantly different between the groups. At M12, 22/61 patients (36%) treated with the standard regimen and 23/46 (50%) with the RA regimen achieved an overall response (R 1 CR). The initial pattern of response at M3 was associated with a later pattern of response by M12 in both groups. In multivariate analysis, both a younger age and a low number of previous therapies were associated with a higher likelihood of overall response at M12. Tolerance was good and comparable between the two groups. The RA regimen is an effective and safe alternative to the standard regimen to treat adults with ITP. Am. J. Hematol. 88:858-861,

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“…Several studies, both in adults and in children, have shown a significant initial response in 40% of the cases and a long-lasting (5 y) response in 20-25% (19). For patients relapsing after rituximab therapy, splenectomy is often the only choice to overcome disease.…”

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confidence: 99%

B-cell hyperfunction in children with immune thrombocytopenic purpura persists after splenectomy

et al. 2015

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Background: Immune thrombocytopenic purpura (ITP) is characterized by reduced platelet count secondary to immunemediated destruction, this results in an increased bleeding risk. Autoantibodies binding to platelets tag them for premature destruction in the spleen. For this reason, splenectomy is often performed as treatment of chronic forms of disease that are resistant to pharmacological therapy. Methods: We studied 30 patients with ITP and compared them with age-matched controls. results: We show that B cells of patients with chronic ITP are intrinsically hyperreactive, producing more than normal IgG in vivo and plasma cells in vitro. In normal individuals after splenectomy, a significant depletion of memory B cells is observed, associated with loss of reactivity to CpG oligodeoxynucleotide and consequent inability to form antibody-producing cells. In Enzyme-Linked ImmunoSpot Methods, we compared three splenectomized ITP patients relapsing after surgery, 30 healthy controls, and 37 individuals splenectomized for trauma, spherocytosis, thalassemia, nonhematological tumor, and other diseases. conclusions: We confirmed that B cells of ITP patients remain hyperreactive in vitro and form high numbers of antibody-producing cells after splenectomy. Thus, chronic ITP may be associated with intrinsic B-cell hyperfunction, leading to the production of antibodies with multiple specificities including that against platelets.

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Outcomes 5 years after response to rituximab therapy in children and adults with immune thrombocytopenia

Cited by 298 publications

References 47 publications

Long‐term follow‐up analysis after rituximab salvage therapy in adult patients with immune thrombocytopenia

Long‐term follow‐up analysis after rituximab salvage therapy in adult patients with immune thrombocytopenia

Efficacy and safety of rituximab given at 1,000 mg on days 1 and 15 compared to the standard regimen to treat adult immune thrombocytopenia

B-cell hyperfunction in children with immune thrombocytopenic purpura persists after splenectomy

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