Definition, diagnosis and treatment of immune thrombocytopenic purpura

George, James N.

doi:10.3324/haematol.2009.007674

Cited by 48 publications

(29 citation statements)

References 18 publications

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“…18 Although a platelet count of Ͻ 30 000/L is often used as a surrogate marker, 4,5,9,19 lower (10-20 000/L) or higher (50 000/L) thresholds are pursued depending on the risk of bleeding, the presence of comorbidities, patient's lifestyle, and risk of trauma, all of which need to be weighed against the probable benefits and the risk of treatmentrelated side effects. 20 Some therapies may improve health-related quality of life, especially fatigue, although this issue is infrequently considered or invoked as a reason to treat. 21,22 Comparative outcome studies are lacking here as well.…”

Section: Who Should Be Treated?mentioning

confidence: 99%

How I treat immune thrombocytopenia: the choice between splenectomy or a medical therapy as a second-line treatment

Ghanima

Godeau

Cines

et al. 2012

Blood

208

157

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The paradigm for managing primary immune thrombocytopenia (ITP) in adults has changed with the advent of rituximab and thrombopoietin receptor agonists (TPO-RAs) as options for second-line therapy. Splenectomy continues to provide the highest cure rate (60%-70% at 5؉ years). Nonetheless, splenectomy is invasive, irreversible, associated with postoperative complications, and its outcome is currently unpredictable, leading some physicians and patients toward postponement and use of alternative approaches. An important predicament is the lack of studies comparing secondline options to splenectomy and to each other. Furthermore, some adults will improve spontaneously within 1-2 years. Rituximab has been given to more than 1 million patients worldwide, is generally well tolerated, and its short-term toxicity is acceptable. In adults with ITP, 40% of patients are complete responders at one year and 20% remain responders at 3-5 years. Newer approaches to using rituximab are under study. TPO-RAs induce platelet counts > 50 000/L in 60%-90% of adults with ITP, are well-tolerated, and show relatively little short-term toxicity. The fraction of TPO-RA-treated patients who will be treatment-free after 12-24 months of therapy is unknown but likely to be low. As each approach has advantages and disadvantages, treatment needs to be individualized, and patient participation in decision-making is paramount. (Blood. 2012;120(5):960-969)

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Section: Who Should Be Treated?mentioning

confidence: 99%

How I treat immune thrombocytopenia: the choice between splenectomy or a medical therapy as a second-line treatment

Ghanima

Godeau

Cines

et al. 2012

Blood

208

157

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“…Splenectomy has long been the "gold standard" second-line treatment for patients with persisting or chronic severe ITP, but it has now become obvious that an increasing number of both clinicians and patients are reluctant to consider/undergo splenectomy. Rituximab has become a possible alternative to splenectomy, although the durability of response and its long-term safety is still a matter of debate and its use in an off-label setting has become a real issue in many countries now that new drugs have been licensed for ITP [41]. The efficacy of TPO-r agonists is unquestionable as clearly shown by several well-designed controlled randomized studies.…”

Section: Resultsmentioning

confidence: 99%

“…They should therefore not be compared and balanced with other treatment strategies such as splenectomy and rituximab which aim to induce long-term remission or even to cure the disease [33]. Therefore, the use of these agents should be restricted to those patients who have a chronic refractory ITP [33,41] according to the recent set of terminology criteria [1]. Whether the transient use of TPO-R agonist could be also helpful for the management of patients with severe persistent ITP still needs to be established as only a minority of these patients will eventually achieve spontaneous remission.…”

Section: Resultsmentioning

confidence: 99%

Treatment of chronic immune thrombocytopenic purpura in adults

Godeau

Michel

2010

Ann Hematol

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Until recently, the management of refractory immune thrombocytopenic purpura (ITP) was a real challenge as shown by the large variety of treatments proposed in both American Society of Hematology and the British Committee for Standards in Haematology guidelines published 10 and 6 years ago, respectively. However, as in the past 5 years, new therapeutic approaches including eradication of Helicobacter pylori in infected patients and rituximab have been proposed and the thrombopoietinreceptor agonists have been developed and licensed for ITP. It is likely that the therapeutic strategy of ITP will be profoundly modified and revisited in the next future. The purpose of this article is to discuss the impact and the place of these new therapeutic options into the whole treatment strategy of chronic ITP and to draw the perspective of new experimental therapies.

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“…[6][7][8] This fact and the lack of evidence that intensive medical therapy administered early in the disease course may improve or even cure ITP 9 mean that some treatments, including rituximab, are not, at present, acceptable as first-line therapy. …”

Section: 3mentioning

confidence: 99%

Comment to the article by Arai Y, Jo T, Matsui H, Kondo T and Takaori-Kondo A: “Comparison of up-front treatments for newly diagnosed immune thrombocytopenia – a systematic review and network meta-analysis”. Haematologica 2018;103(1):163–171. Need to direct immune thrombocytopenia therapy towards shared goals

Cirasino¹,

Semeraro²

2018

Haematologica

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On the basis of the results of their recent work titled "Comparison of up-front treatments for newly diagnosed immune thrombocytopenia -a systematic review and network meta-analysis", Arai and colleagues sustain that human thrombopoietin agonists may be beneficial up-front therapies in addition to conventional corticosteroid monotherapy in adults with primary immune thrombocytopenia, and that head-to-head trials including these regimens and rituximab-containing treatments are necessary to determine the most suitable therapies for these patients. Such head-to-head trials would, however, clash with the treatment currently foreseen for adults with newly diagnosed primary and severe immune thrombocytopenia which is not actually directed at modifying the natural history of the disease, but at 'achieving, rapidly, a safe platelet count to prevent or stop hemorrhages and to ensure an acceptable quality of life, avoiding, as much as possible, treatment-related adverse effects'. This fact and the lack of evidence that intensive medical therapy administered early in the disease course may improve or even cure immune thrombocytopenia mean that some treatments, including rituximab, are not, at present, acceptable as first-line therapy. In consideration of the potential usefulness of thrombopoietin agonists in up-front treatment for newly diagnosed immune thrombocytopenia, head-to-head trials similar to those proposed by Arai and colleagues would require a preliminary agreement on the goal of this line of therapy, just as we have sustained for other lines of immune thrombocytopenia therapy. This is to prevent exacerbating the disagreements already existing between different guidelines on the treatment of immune thrombocytopenia.In a recent article in Haematologica, Arai and colleagues reported the results of a systematic review and network meta-analysis comparing the efficacy and safety of different treatments utilized in adults for primary newly diagnosed immune thrombocytopenia (ITP). 1The study found that the main outcome, represented by a sustained response (platelet count >30x10 9 /L for 3-6 months after completion of treatments), was achieved by a significantly higher proportion of patients treated with recombinant human thrombopoietin agonists + dexamethasone or rituximab + dexamethasone than by those treated with conventional prednisolone or dexamethasone monotherapy. Moreover, with regards to the two secondary endpoints considered, overall response (defined as a platelet count >30x10 9 /L for 2-4 weeks after initiation of the upfront treatment) and therapy-related adverse events, thrombopoietin agonists + dexamethasone and thrombopoietin agonists + prednisolone improved early overall responses compared to prednisolone, dexamethasone, and rituximabcontaining regimens, while all treatments were tolerated and the therapy-related adverse event profiles were similar in all treatment arms. 1These results led the authors to claim that thrombopoietin agonists may be beneficial up-front therapies in addition to conventional ...

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Definition, diagnosis and treatment of immune thrombocytopenic purpura

Cited by 48 publications

References 18 publications

How I treat immune thrombocytopenia: the choice between splenectomy or a medical therapy as a second-line treatment

How I treat immune thrombocytopenia: the choice between splenectomy or a medical therapy as a second-line treatment

Treatment of chronic immune thrombocytopenic purpura in adults

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