How I treat idiopathic thrombocytopenic purpura (ITP)

Cines, Douglas B.; Bussel, James B.

doi:10.1182/blood-2004-12-4598

Cited by 316 publications

(290 citation statements)

References 89 publications

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“…A gyógyszer jól tolerálható, hosszan tartó kezelés mellett is csak ritkán jelentkeznek enyhe mellékha-tások (májfunkciózavar, leukopenia). A többi számításba jövő készítmény ebben az életkorban és indikációban nincsen törzskönyvezve -vinkrisztin 0,02 mg/kg iv., hetente egyszer, négy-hat alkalommal; ciklosporin-A 2,5-3,0 mg/kg/nap per os; ciklofoszfamid (2 mg/kg/nap ÖSSZEFOGLALÓ KÖZLEMÉNY per os), merkaptopurin (25-50 mg/m 2 ) -, és egyre kevésbé is használjuk őket [10]. Az újabb irodalmi adatok és egyes ajánlások már az új innovatív TPO-stimuláló készítményeket is a másodvonalban említik (l. később).…”

Section: Második Vonalbeli Kezelésunclassified

Újdonságok a gyermekkori immunthrombocytopenia kezelésében – 2017

Kovács

Kiss

2017

Orvosi Hetilap

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A gyermekkori immunthrombocytopenia (ITP) nagyon változatos kórkép. A különböző nemzetközi ajánlások nem szabnak meg szigorú előírásokat, csupán terápiás lehetőségekről beszélnek. A hazai gyakorlat alapján 2011-ben egy konkrét terápiás algoritmus került bemutatásra. Az elmúlt években új innovatív szerek állnak rendelkezésre már Magyarországon is, ezért időszerű a terápiás ajánlások átgondolása és újrafogalmazása. A jelen munkában napjaink korszerű ellátási elveit mutatjuk be. Csecsemő-és gyermekkorban továbbra is elsősorban a nagy adagú intravénás immunglobulin-kezelést javasoljuk, idősebb életkorban és alternatívaként a szteroidkezelés is indikált (lökéskezelés vagy elnyújtott kis adagú kezelés formájában). Rezisztens esetekben az új, innovatív trombopoetinreceptor-izgató szerek közül ma már gyermekeknek is törzskönyvezett készítmény az eltrombopág, amely nagyon hatékonyan javítja a kór-lefolyást. A lépkivétel gyermekkorban csak kivételes esetben jön szóba. Az ITP kiszámíthatatlan kórkép, és bár gyermekkorban mintegy 70-80%-ban gyógyul, szakorvosi ellátást és hosszú távú követést igényel. Orv Hetil. 2017; 158(48): 1891-1896. Kulcsszavak: gyermekkori ITP, vérzés, terápiás ajánlás, immunglobulin, trombopoetinreceptor Novelties in the treatment of pediatric immune thrombocytopenia -2017Immune thrombocitopenia in children is a very variable disease. International recommendations give therapeutic possibilities without strong protocols. In 2011, a therapeutic algorithm was published based on Hungarian practice. Recently, new innovative drugs have been available even in Hungary, so there is a need for modification of the therapeutic protocols. In this summary we give an overview about the current up-to-date management. In infancy and in childhood, high-dose immunglobulin treatment is recommended henceforward. In older children an alternative can be steroid therapy (pulses or long-term low-dose treatment). In resistant cases, a new thrombopoetin receptor stimulant, eltrombopag can be administered. This drug is registered in Hungary, and can very effectively influence the prognosis. Splenectomy is very rare nowadays in children. Immune thrombocytopenia is an unpredictable disease. Cure rate is about 70-80% of the cases, but management of the patients needs special care and specialist.

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Section: Második Vonalbeli Kezelésunclassified

Újdonságok a gyermekkori immunthrombocytopenia kezelésében – 2017

Kovács

Kiss

2017

Orvosi Hetilap

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“…In adults, the incidence is higher in women than in men [2], whereas the reverse is true in children, where ITP predominates in young male [7]. Clinical presentation varies from asymptomatic or mild symptoms (bruising and petechiae) to mucocutaneous bleeding that can become severe [5,8].…”

Section: Introductionmentioning

confidence: 99%

Eltrombopag: an update on the novel, non-peptide thrombopoietin receptor agonist for the treatment of immune thrombocytopenia

Kühne

Imbach

2010

Ann Hematol

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Immune thrombocytopenia (ITP) is characterised by a transient or persistent decrease in platelets accompanied by an increased risk of bleeding, which can have a significant negative impact on patients' health-related quality of life. The condition has long been associated with an increased rate of immune-mediated platelet destruction, and traditional treatments have targeted the reduction in platelet destruction; however, some interventional drugs are limited by transient efficacy and side effects. Recent advances in our understanding of ITP pathogenesis have highlighted the role of impaired platelet production, which has led to the advent of a new generation of thrombopoietin (TPO)-receptor agonist therapies, including eltrombopag and romiplostim. The oral, non-peptide TPO-receptor agonist eltrombopag has shown considerable promise in both preclinical and clinical trials. Eltrombopag has a unique mechanism of action and binds to a transmembrane region of the TPO receptor that is distant from the TPO binding site. As such, eltrombopag may confer synergistic effects with endogenous TPO rather than competing for binding. Eltrombopag also induces activation of the TPO receptor and downstream signalling in a distinct manner to TPO and does not have a significant impact on platelet function. Clinical evidence demonstrates that eltrombopag produces a rapid and sustainable increase in platelet counts that significantly reduces bleeding and is well tolerated in patients with ITP. Eltrombopag therefore represents an important addition to the therapeutic armamentarium for ITP.

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“…Rarely, life-threatening bleeding occurs, with intracranial hemorrhage being the principle cause of death [1,7]. Serious hemorrhage is most likely to occur when the platelet count falls below (10-20) Â 10 9 /L [1,[7][8][9]. Therefore, initial treatment options for ITP are intended to rapidly increase platelet counts and include glucocorticoids, immune globulin intravenous (IGIV), and intravenous anti-Rho (D) globulin in Rho (D)-positive, DATnegative patients [9].…”

Section: Introductionmentioning

confidence: 99%

“…Serious hemorrhage is most likely to occur when the platelet count falls below (10-20) Â 10 9 /L [1,[7][8][9]. Therefore, initial treatment options for ITP are intended to rapidly increase platelet counts and include glucocorticoids, immune globulin intravenous (IGIV), and intravenous anti-Rho (D) globulin in Rho (D)-positive, DATnegative patients [9]. The first report of the efficacy of IGIV in the treatment of ITP appeared in 1981 [10].…”

Section: Introductionmentioning

confidence: 99%

Safety and tolerability of a novel chromatography‐based intravenous immunoglobulin when administered at a high infusion rate in patients with immune thrombocytopenic purpura

Bussel

Kim

2006

American J Hematol

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Intravenous immunoglobulin (IGIV) therapy is generally considered to be a safe and effective treatment for idiopathic thrombocytopenic purpura (ITP). The usual initial treatment dose is 1-2 g/kg body weight, which results in an extended infusion time, significantly impacting patients' day-to-day activities. Two crossover studies assessed the safety and tolerability of a novel IGIV preparation (IGIV-C; Gamunex 1 , 10%) when infused at rates ranging from 0.08 mL/kg/min (the standard maximum licensed rate) to 0.14 mL/kg/min in patients with ITP. The first study included 28 patients and 3 infusion rates; 0.08, 0.11, and 0.14 mL/kg/min. The second study included 8 patients and 2 infusion rates; 0.08 and 0.14 mL/kg/min were evaluated. The incidence of infusion-related adverse events was similar for all infusion rates. Headache was the most commonly reported infusion-related adverse event. The incidence, combined for Studies 1 and 2, was 14.7% (n 5 34), 18.2% (n 5 22), and 19.4% (n 5 31) of patients, for each infusion rate of 0.08, 0.11, and 0.14 mL/kg/min, respectively. The majority were mild in severity. None of the other drug-related, treatment-emergent events were serious; most were mild, in spite of the higher rate of fluid loading over a shorter period of time for patients infused at 0.14 mL/kg/min. There were no clinically important changes in parameters that distinguished between infusion rates; there were no signs of hemolysis. The results suggest that IGIV-C infused at rates up to 0.14 mL/kg/min in patients with ITP is well tolerated. Am. J. Hematol. 82:192-198, 2007. V V C 2006 Wiley-Liss, Inc.

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How I treat idiopathic thrombocytopenic purpura (ITP)

Cited by 316 publications

References 89 publications

Újdonságok a gyermekkori immunthrombocytopenia kezelésében – 2017

Újdonságok a gyermekkori immunthrombocytopenia kezelésében – 2017

Eltrombopag: an update on the novel, non-peptide thrombopoietin receptor agonist for the treatment of immune thrombocytopenia

Safety and tolerability of a novel chromatography‐based intravenous immunoglobulin when administered at a high infusion rate in patients with immune thrombocytopenic purpura

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