Update on the management of immune thrombocytopenic purpura in children

Tarantino, Michael D.

doi:10.1097/moh.0b013e3282ab98df

Cited by 29 publications

(27 citation statements)

References 63 publications

(50 reference statements)

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“…In fact, at present, there is a lack of randomized trials focusing also on bleeding, quality of life, adverse effects, and costs. 13,14 The administration of pulsed high-dose dexamethasone (HD-DXM) at a dose of 40 mg/d given orally according to a 4-day course repeated each 28 days for 6 consecutive times in adult ITP refractory to several therapy lines, dates back to the mid-1990s. Indeed, in a small cohort of patients a response rate of 100% has been observed, 15 but in other studies, the same approach was not so successful.…”

Section: Introductionmentioning

confidence: 99%

Therapy with high-dose dexamethasone (HD-DXM) in previously untreated patients affected by idiopathic thrombocytopenic purpura: a GIMEMA experience

Mazzucconi

Fazi

Bernasconi

et al. 2006

Blood

232

181

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In idiopathic thrombocytopenic purpura (ITP), corticosteroids have been widely recognized as the most appropriate firstline treatment, even if the best therapeutic approach is still a matter of debate. Recently, a single high-dose dexamethasone (HD-DXM) course was administered as first-line therapy in adult patients with ITP. In this paper we show the results of 2 prospective pilot studies (monocentric and multicentric, respectively) concerning the use of repeated pulses of HD-DXM in untreated ITP patients. In the monocenter study, 37 patients with severe ITP, age at least 20 years and no more than 65 years, were enrolled. HD-DXM was given in 4-day pulses every 28 days, for 6 cycles. Response rate was 89.2%; relapse-free survival (RFS) was 90% at 15 months; long-term responses, lasting for a median time of 26 months (range 6-77 months) were 25 of 37 (67.6%). In the multicenter study, 95 patients with severe ITP, age at least 2 years and no more than 70 years, were enrolled. HD-DXM was given in 4-day pulses every 14 days, for 4 cycles; 90 patients completed 4 cycles. Response rate (85.6%) was similar in patients classified by age (< 18 years, 36 of 42 ‫؍‬ 85.7%; > 18 years, 41 of 48 ‫؍‬ 85.4%, P ‫؍‬ not significant), with a statistically significant difference between the second and third cycle (75.8% vs 89%, P ‫؍‬ .018). RFS at 15 months 81%; long-term responses, lasting for a median time of 8 months (range 4-24 months) were 67 of 90 (74.4%). In both studies, therapy was well tolerated. A schedule of 3 cycles of HD-DXM pulses will be compared with standard prednisone therapy (eg, 1 mg/kg per day) in the next randomized Gruppo Italiano Malattie EMatologiche dell'Adulto (GIMEMA) trial. (Blood.

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Section: Introductionmentioning

confidence: 99%

Therapy with high-dose dexamethasone (HD-DXM) in previously untreated patients affected by idiopathic thrombocytopenic purpura: a GIMEMA experience

Mazzucconi

Fazi

Bernasconi

et al. 2006

Blood

232

181

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“…A kezelés célja a súlyos, potenciálisan fatális vérzések megelőzése és a lehetőség szerinti korlátozásmentes élet-mód biztosítása jelentős terápiás mellékhatások és szö-vődmények elkerülésével [5][6][7][8][9] …”

Section: 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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“…This review will briefly discuss current practice in the diagnosis and management of acute and chronic ITP of childhood, as basics of this disease are well known to practicing hematologists and several comprehensive reviews of current practice have recently been published. [1][2][3] The remainder of this article will examine new insights into pathophysiology of immune thrombocytopenia in children, including neonatal alloimmunization, as well as novel therapeutic approaches currently in use and those emerging on the horizon.…”

Section: Diane J Nugentmentioning

confidence: 99%

“…[1][2][3] For decades, physicians and researchers have commented on the rapid onset of bruising and mucosal bleeding in a severely thrombocytopenic child with minimal or no trauma. 4 The remainder of the complete blood count and physical exam should be normal.…”

Section: Diagnosismentioning

confidence: 99%

Immune Thrombocytopenic Purpura of Childhood

Nugent¹

2006

Hematology

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Immune mediated thrombocytopenia (ITP) is a common manifestation of autoimmune disease in children. Although patients often present with bruises, petechiae, and some mucosal bleeding, the incidence of life-threatening hemorrhage is rare (0.2-0.9%) but can be fatal when presenting in vital organs. A wide range of therapeutic regimens are currently in use, including observation alone, as the majority of children recover within 4-6 months regardless of treatment. A growing understanding of the pathophysiology of acute ITP in children has not impacted the controversy surrounding treatment, but has clarified the mechanism of action of the most frequently used agents in chronic ITP. Newer monoclonal antibodies such as Rituxan have proved very useful in chronic or refractory ITP and studies are ongoing to determine the best regimens using this form of immune modulation. Splenectomy and newer agents to boost platelet production are also under study in chronic ITP. Neonates may also have a form of immune thrombocytopenia with extensive bruising and thrombocytopenia called neonatal alloimmune thrombocytopenic purpura (NATP). Rather than autoantibodies, the platelet destruction is secondary to transplacental maternal IgG alloantibodies. During pregnancy mothers may become sensitized to platelet membrane antigens present on fetal platelets. These antibodies may result in serious bleeding, including intracranial hemorrhage in the perinatal period. Once identified, these mothers may require treatment during future pregnancies to minimize serious bleeding in the fetus and neonate. Treatment in utero and immediately following delivery is focused on restoring neonatal platelets to a safe level and preventing life-threatening bleeding.

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Update on the management of immune thrombocytopenic purpura in children

Cited by 29 publications

References 63 publications

Therapy with high-dose dexamethasone (HD-DXM) in previously untreated patients affected by idiopathic thrombocytopenic purpura: a GIMEMA experience

Therapy with high-dose dexamethasone (HD-DXM) in previously untreated patients affected by idiopathic thrombocytopenic purpura: a GIMEMA experience

Újdonságok a gyermekkori immunthrombocytopenia kezelésében – 2017

Immune Thrombocytopenic Purpura of Childhood

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