Initial Treatment of Immune Thrombocytopenic Purpura with High-Dose Dexamethasone

Cheng, Yunfeng; Wong, Raymond Siu Ming; Soo, Yannie; Chui, Chung Hin; Lau, Fung Yi; Chan, Natalie Pui Ha; Wong, Wai Keung; Cheng, Gregory

doi:10.1056/nejmoa030254

Cited by 280 publications

(239 citation statements)

References 20 publications

(28 reference statements)

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“…We have obtained a persistent response rate of 66.7% (32/48 evaluable adult patients) without any therapy, whereas in the Cheng et al study a persistent response rate of 42% was reported. 21 Furthermore, our data seem to be similar to those of Borst et al, 23 who more recently reported a long-term response of 59%. However, they observed an adverse event rate of 22%, and in our 2 studies adverse event rates were 13.5% and 2.1%, respectively.…”

Section: Discussionsupporting

confidence: 91%

“…The results obtained by the GIMEMA multicenter pilot study in adults are comparable to those recently reported by Cheng et al 21 for the initial response rate (85% vs 85%). However, the relapse rate of 22% observed in the GIMEMA study is very different from that observed by Cheng et al, who reported a 50% relapse rate within 6 months from response achievement.…”

Section: Discussionsupporting

confidence: 88%

“…Moreover, all relapsed patients had a response to a second therapy course, but only a minority of them (18.5%) showed a persistent response after discontinuation of maintenance therapy. 21 Although it seems that HD-DXM has a better response rate than conventional prednisone doses, we do not know whether results could be improved by reducing relapse rate, and thus improving persistent responses, as there are no randomized studies.…”

Section: Introductionmentioning

confidence: 98%

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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: Discussionsupporting

confidence: 91%

Section: Discussionsupporting

confidence: 88%

Section: Introductionmentioning

confidence: 98%

See 1 more Smart Citation

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

View full text Add to dashboard Cite

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“…A previous study also showed that Dex monotherapy was related to a higher incidence of posttherapeutic relapse (50%), although the initial remission was high (85%). 15 Late relapse is one of the critical problems in ITP treatment, [1][2][3][4] and there is an ongoing need to develop strategies to suppress relapse. Our findings indicated that additional courses of Dex (two to six total courses) and/or tapering PSL could significantly suppress relapse (21%) compared with PSL (37%).…”

Section: Discussionmentioning

confidence: 99%

Untitled

2017

TH Open

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“…Childhood ITP is typically a disease of acute onset; more than 80% of children will have a spontaneous and permanent remission within 1 year of onset [2]. In contrast, adults tend to have more persistent ITP [3], although recent studies suggest that there is a tendency for certain patients to improve with time [3][4][5][6]. Rarely, life-threatening bleeding occurs, with intracranial hemorrhage being the principle cause of death [1,7].…”

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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Initial Treatment of Immune Thrombocytopenic Purpura with High-Dose Dexamethasone

Abstract: A four-day course of high-dose dexamethasone is effective initial therapy for adults with immune thrombocytopenic purpura.

Cited by 280 publications

References 20 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

Untitled

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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