Stasi, R., Pagano, A., Stipa, E. & Amadori, S. Rituximab chimeric anti-CD20 monoclonal antibody treatment for adults with chronic idiopathic thrombocytopenic purpura. Blood 98, 952-957

University of Rome Tor Vergata, Roma, Latium, Italy
Blood (Impact Factor: 10.45). 09/2001; 98(4):952-7. DOI: 10.1182/blood.V98.4.952
Source: PubMed


The role of rituximab, a chimeric monoclonal antibody directed against the CD20 antigen, in the treatment of patients with chronic idiopathic thrombocytopenic purpura (ITP) has not been determined. The effectiveness and side effects of this therapeutic modality were investigated in a cohort of 25 individuals with chronic ITP. All patients had ITP that had been resistant to between 2 and 5 different therapeutic regimens, including 8 patients who had already failed splenectomy. Patients were scheduled to receive intravenous rituximab at the dose of 375 mg/m(2) once weekly for 4 weeks. Rituximab infusion-related side effects were observed in 18 patients, but were of modest intensity and did not require discontinuation of treatment. A complete response (platelet count greater than 100 x 10(9)/L) was observed in 5 cases, a partial response (platelet count between 50 and 100 x 10(9)/L) in 5 cases, and a minor response (platelet count below 50 x 10(9)/L, with no need for continued treatment) in 3 cases, with an overall response rate of 52%. In 7 cases, responses were sustained (6 months or longer). In 2 patients with relapsed disease, repeat challenge with rituximab induced a new response. In patients with a complete or partial response, a significant rise in platelet concentrations was observed early during the course of treatment, usually 1 week after the first rituximab infusion. No clinical or laboratory parameter was found to predict treatment outcome, although there was a suggestion that women and younger patients have a better chance of response. In conclusion, rituximab therapy has a limited but valuable effect in patients with chronic ITP. In view of its mild toxicity and the lack of effective alternative treatments, its use in the setting of chronic refractory ITP is warranted. (Blood. 2001;98:952-957)

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    • "Dans les formes chroniques avec une thrombopénie profonde, la splénectomie est une thérapeutique de choix. Stasi et al. ont rapporté en 2001 25 cas de PTI chroniques ayant reç u préalablement 2 à 5 lignes thérapeutiques, 8 ayant été splénectomisés [27]. Ces patients ont été traités par rituximab selon les modalités utilisées dans le LNH (375 mg/m 2 ). "
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    ABSTRACT: Le rituximab est un anticorps (Ac) monoclonal chimérique qui cible l’antigène CD20 à la surface des lymphocytes B (LB). Il induit une destruction sélective des LB, qu’ils soient normaux ou éventuellement tumoraux, et a une efficacité démontrée dans le traitement des patients ayant un lymphome non-Hodgkinien (LNH) B, indication dans laquelle ce médicament à l’autorisation de mise sur le marché (AMM) aux États-Unis depuis 1997. Depuis 2001, l’efficacité du rituximab a été étudiée dans le traitement d’un large éventail de pathologies auto-immunes. Parmi ces dernières, on trouve en particulier la polyarthrite rhumatoïde (PR) [1,2], le purpura thrombopénique immunologique (PTI) [3], le lupus érythémateux systémique (LES) [4] et les vascularites associées aux anticorps anti-cytoplasme de polynucléaire neutrophile (ANCA) pour n’en citer que quelques unes. Dans cette revue générale nous ne pourrons pas aborder l’ensemble des pathologies autoimmunes systémiques au cours desquelles le rituximab a été étudié (Tableau 1) et nous nous limiterons à son utilisation au cours du lupus systémique, des cytopénies auto-immunes, des microangiopathies thrombotiques (MAT) et des vascularites.
    Full-text · Article · Nov 2014 · La Revue de Médecine Interne
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    • "Early anti-CD20 data outcome is also inferior to outcomes seen after splenectomy. However, all but two published studies (Stasi et al, 2001; Cooper et al, 2004) have reported only a small number of patients, and most cohorts have included a mix of splenectomized and/or non-splenectomized patients. Although a recent French pilot study assessed rituximab efficacy and safety in non-splenectomized adults with chronic ITP (Godeau et al, 2008), it remains important to determine whether rituximab is an effective treatment for delaying or avoiding splenectomy in chronic ITP. "
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    ABSTRACT: Primary immune thrombocytopenia (ITP) is an acquired immune-mediated disorder with absence of any underlying cause. Corticosteroids are the standard initial treatment. Splenectomy is the main second-line treatment. A trend to delay or avoid splenectomy has developed thanks to new agents like rituximab. Few studies have assessed the response rate to rituximab in chronic ITP. We performed the first meta-analysis of randomized clinical trials and observational studies on rituximab as an effective splenectomy-avoiding option in adult chronic ITP. Overall methods were adapted from published guidelines for meta-analysis (meta-analysis of observational studies in epidemiology and preferred reporting items for systematic reviews and meta-analyses). Two haematologist investigators carried out study selection and data extraction independently, recording overall response rate (ORR) and complete response (CR) as primary assessment criteria. Of 364 records were identified through electronic databases. Of 19 retrospective or prospective observational studies were retained after removing duplicate studies and full-text analyses. The ORR was 57% (95% confidence interval [CI]: 48-65), for 368 non-splenectomized patients after rituximab; CR was 41% (95% CI: 0·33-0·51) for 346 patients. Results were stable for ORR and CR in all sub-analyses. In univariate or multivariate mixed-effect meta-regression, age was the most relevant effect. According to our results, rituximab should be used in earlier in non-splenectomized patients.
    Full-text · Article · May 2012 · British Journal of Haematology
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    • "However, safety concerns have also been noted [11,14]. Although standard and emerging therapies have reduced the risk of bleeding among chronic ITP patients, treatments are associated with side effects that may impose substantial burden on patients. "
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    ABSTRACT: Chronic immune thrombocytopenia (ITP) is a debilitating autoimmune disorder that causes a reduction in blood platelets and increased risk of bleeding. ITP is currently managed with various pharmacologic therapies and splenectomy.This study was conducted to assess patient perceived and reported treatment side effects, as well as the perceived burden or bother, and need to reduce or stop treatment, associated with these side effects among adult patients with chronic ITP. A Web-enabled survey was administered to members of a US-based ITP patient support group. Patients reported demographic and clinical characteristics, ITP treatments' side effects for treatments received since diagnosed, level of bother (or distress), and need to reduce or stop treatment, associated with side effects. Current and past exposure was assessed for five specific treatment types: corticosteroids (CS), intravenous immunoglobulin (IVIg), anti-D immunoglobulin (anti-D), rituximab (RT), and splenectomy (SPL), as well as for other patient-referenced therapies (captured as "other"). The survey was completed by 589 patients; 78% female, 89% white, mean age 48 years (SD = 14.71), and 68% reported a typical low platelet count of < 50,000/μL. Current or past treatment with CS was reported by 92% (n = 542) of patients, 56% (n = 322) for IVIg, 36% (n = 209) for anti-D, 36% (n = 213) for RT, and 39% (n = 227) for SPL. A substantial proportion of CS-treated patients reported side effects (98%, P < 0.05), were highly bothered by their side effects (53.1%, P < 0.05), and reported the need to stop or reduce treatment due to side effects (37.8%, P < 0.05). Among patients reporting side effects of treatment, significant associations were noted for the number of side effects, aggregate bother of reported side effects, and the need to stop or reduce treatment (all P < 0.05). Current ITP treatments, particularly corticosteroids, are associated with multiple bothersome side effects that may lead to patients stopping or reducing therapy. Open, informed and complete communication between clinician and patient regarding both the benefits and the side effects of ITP treatment may better prepare patients for their prescribed regimens.
    Full-text · Article · Mar 2012 · BMC Blood Disorders
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