Efficiency of curative and prophylactic treatment with rituximab in ADAMTS13-deficient thrombotic thrombocytopenic purpura: a study of 11 cases

Fakhouri, Fádi; Vernant, Jean Paul; Veyradier, Agnès; Wolf, Martine; Kaplanski, Gilles; Binaut, R.; Rieger, Manfred; Scheiflinger, Friedrich; Poullin, Pascale; Deroure, Benjamin; Delarue, Richard; Lesavre, Philippe; Vanhille, Philippe; Hermine, Olivier; Remuzzi, Giuseppe; Grünfeld, J. P.

doi:10.1182/blood-2005-03-0848

Cited by 257 publications

(245 citation statements)

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“…A median follow-up of nearly 50 months is reflecting the longest follow-up period after rituximab treatment reported to date. In 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20 21 22 23 24 25 26 27 28 29 30 31 32 33 34 35 36 37 38 39 40 41 42 43 44 45 46 47 48 49 50 51 52 53 54 55 56 57 58 59 60 61 62 63 64 65 the existing literature patients treated with plasma exchange and rituximab have been followed for up to three years [17,18], however median follow-up is reported less than 11 months [12]. Longer follow-up is required for addressing possible long-term side effects of rituximab and the safety of this treatment procedure [24].…”

Section: Resultsmentioning

confidence: 99%

“…Using this regimen, although only a small number of patients have been reported worldwide, the majority of patients with refractory or relapsing TTP achieved complete remission with complete clinical and laboratory responses including normal ADAMTS13 level and disappearance of anti-ADAMTS13 antibodies [11]. In the reported cases, only 10% -13% of patients relapsed after rituximab treatment after a median follow-up of 11 months [17][18]. Especially when considering B-cell recovery nine to twelve months after rituximab application, data for longterm follow-up is required.…”

Section: Word Countsmentioning

confidence: 99%

“…In case of plasmarefractory TTP, which is defined as persistent thrombocytopenia or lactate dehydrogenase elevation after a total of seven daily plasma exchange procedures, current guidelines advise to intensify plasma exchange and to add corticosteroids in patients not receiving immunosuppressive drugs [12]. Due to the autoimmune nature of acquired idiopathic TTP rituximab has been used as a second-line immunosuppressive intervention in TTP cases refractory to plasma exchange as well as in frequently relapsing patients [16][17][18][19]. According to treatment schedules in Non-Hodgkin's lymphoma, rituximab is mainly administered at doses of 375 mg/m 2 weekly for an average of 4 doses.…”

Section: Word Countsmentioning

confidence: 99%

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Long-term follow-up of idiopathic thrombotic thrombocytopenic purpura treated with rituximab

et al. 2010

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Rituximab may be used to treat patients with thrombotic thrombocytopenic purpura (TTP) refractory to plasma exchange or recurrent disease. While initial response rates are reported to be high, long-term follow-up data of patients treated with rituximab are not available to date however important to estimate the safety and benefit of this treatment. 12 patients with non-familial idiopathic TTP refractory to plasma exchange or with recurrent disease treated with rituximab between 2000 and 2008 were re-examined. The median follow-up was 49.6 months, ranging from 11 to 97 months. All patients achieved initial complete remission after application of rituximab. During follow-up, nine patients remained disease-free and three patients suffered from recurrent disease. All patients with recurrent disease responded to subsequent rituximab therapy. No long-term side effects were noted during the follow-up period. In conclusion, rituximab represents an effective second-line treatment option in relapsing or refractory TTP. Still, patients need to be closely monitored for relapses with extended follow-up.Response to Reviewers: Reviewer #11. Abstract, line 2: I think "acceptable" is not appropriate until larger studies are done. I would suggest changing to: "Rituximab may be used to treat relapsed or refractory TTP" Changes were made accordingly. 2.Abstract, line 5: While initial response rates are "reported to be" high..Changes were made accordingly.3. Abstract, line 32: "Still, patients need to be closely monitored as the relapse rate increases with longer follow-up." Change to ".monitored for relapses with extended follow-up".Changes were made accordingly. 4.Introduction, line 41: Remove "not otherwise explained"Changes were made accordingly. 5.Introduction, line 51: Revise: "Acquired idiopathic TTP is differentiated from hereditary TTP (Upshaw-Schulman syndrome)." ie how are they differentiated?The differentiation between acquired and congenital TTP follows in detail in the next paragraph: "Large progress was made in recent years in understanding the pathophysiology of TTP. Moake et al. described unusually large von Willebrand factor (VWF) multimers in the plasma of patients with relapsing acquired or congenital TTP causing intravascular platelet aggregation occluding the microvasculature [2]. Tsai et al. [3] and Furlan et al. [4] independently identified a VWF-cleaving protease (ADAMTS13) missing from the plasma of patients with congenital TTP [5] and severely deficient in patients with acquired idiopathic TTP. Mutations in the ADAMTS13 gene were shown to cause congenital TTP [6], whereas IgG autoantibodies inhibit the enzyme in most cases of the acquired form of the disease [7][8]." To avoid repetitions we left this part of the introduction as it was. If the editor has a distinct opinion to this point, we are more than willing to change it. 6.Introduction, line 12 (2nd page of introduction) and throughout the paper: "plasma exchange against fresh frozen plasma" change to ". WITH fresh frozen plasma."Changes w...

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Section: Resultsmentioning

confidence: 99%

Section: Word Countsmentioning

confidence: 99%

Section: Word Countsmentioning

confidence: 99%

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Long-term follow-up of idiopathic thrombotic thrombocytopenic purpura treated with rituximab

et al. 2010

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“…Prospective studies and smaller case series have successfully and safely used rituximab, usually as a second-line therapy in patients with acute TTP who fail to respond to standard daily PEX and steroids or in relapsed acute idiopathic TTP patients who have previously demonstrated antibody to ADAMTS13 and electively choose to take rituximab to prevent further relapses. [20][21][22] However, no study has demonstrated the safety or efficaciousness of rituximab given at the time diagnosis.…”

Section: Discussionmentioning

confidence: 99%

A phase 2 study of the safety and efficacy of rituximab with plasma exchange in acute acquired thrombotic thrombocytopenic purpura

Scully

McDonald

Cavenagh

et al. 2011

Blood

376

388

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The safety and efficacy of weekly rituximab 375 mg/m 2 (؋4), given within 3 days of acute TTP admission, with standard therapy (PEX and steroids) was evaluated. Clinical outcomes were compared to historical controls (n ‫؍‬ 40) who had not received rituximab. Within the trial group, 15 of 40 required ICU admission and 15% of all cases with the highest troponin T levels on admission were ventilated. Before the second rituximab infusion, 68% of cases had a platelet count > 50 ؋ 10 9 /L and 38% > 150 ؋ 10 9 /L. Fewer PEX were required in whites compared to nonwhite in the rituximab group (mean 14 vs 21, P ‫؍‬ .0095). Inpatient stay was reduced by 7 days in the non-ICU trial cases compared to historical controls (P ‫؍‬ .04), especially in whites, with a mean reduction of 7 days (P ‫؍‬ .05). Ten percent of trial cases relapsed, median, 27 months (17-31 months), compared to 57% in historical controls, median 18 months (3-60 months; P ‫؍‬ .0011).There were no excess infections or serious adverse events with rituximab. In conclusion, rituximab appears a safe and effective therapy. Inpatient stay and relapse are significantly reduced in the rituximab cohort. Rituximab should be considered in conjunction with standard therapy on acute presentation of TTP. This study was registered at www.clinicaltrials.gov as NCT009-3713. (Blood. 2011;118(7): 1746-1753)

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“…Quando a plasmaférese não está disponível, o paciente deve iniciar a terapia com corticosteroides objetivando a redução da produção de anticorpos antiplaquetários. 40 A troca do plasma de maneira empírica está preconizada como tratamento primário para PTT idiopática e reduz a mortalidade aguda de mais de 90% para menos de 20%. Os critérios para selecionar estes pacientes incluem anemia hemolítica microangiopática, trombocitopenia e ausência de outra explicação óbvia para a PTT, como câncer, sepse, coagulação intravascular disseminada, transplante de tecidos, uso de determinadas drogas e diarreia sanguinolenta recente.…”

Section: Tratamentounclassified

Púrpura trombocitopênica trombótica: o papel do fator von Willebrand e da ADAMTS13

Tonaco¹,

Rios²,

Vieira³

et al. 2010

Rev. Bras. Hematol. Hemoter.

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Efficiency of curative and prophylactic treatment with rituximab in ADAMTS13-deficient thrombotic thrombocytopenic purpura: a study of 11 cases

Cited by 257 publications

References 34 publications

Long-term follow-up of idiopathic thrombotic thrombocytopenic purpura treated with rituximab

Long-term follow-up of idiopathic thrombotic thrombocytopenic purpura treated with rituximab

A phase 2 study of the safety and efficacy of rituximab with plasma exchange in acute acquired thrombotic thrombocytopenic purpura

Púrpura trombocitopênica trombótica: o papel do fator von Willebrand e da ADAMTS13

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