A trial of high-dose dexamethasone therapy for chronic idiopathic thrombocytopenic purpura in childhood☆,☆☆,★
Section snippets
METHODS
Seventeen patients (10 girls, 7 boys) aged between 4 and 17 years (median, 10 years) who had ITP of 6 months' to 14 years' duration were enrolled in this study. These represented the entire population of patients with symptomatic chronic ITP followed at our institutions, for whom no contraindication to the use of dexamethasone existed. Diagnosis had been made on clinical grounds, in accordance with established criteria. In all cases a bone marrow aspirate had shown normal to increased numbers
RESULTS
One month after the end of therapy, six patients (35% of the total) had platelet counts within the normal range. In six children the platelet count increased at the end of each of the six courses but rapidly returned to the pretreatment values. One patient discontinued therapy after the fourth course and four after the fifth because of lack of response, associated in one case with severe side effects. One year after the end of therapy, five of the six responders (29% of the total) still had
DISCUSSION
Complete success was recently reported in treating 10 adults affected by chronic ITP with brief, repeated courses of dexamethasone therapy, given under the assumption that this drug at the high dosage employed is capable of suppressing or even eradicating the population of plasma cells responsible for the continued production of anti-platelet antibody and hence of curing chronic ITP. 4 These encouraging results prompted us to start a trial of the same treatment in pediatric patients. We chose
References (7)
- et al.
Idiopathic thrombocytopenia, initial illness and long-term follow-up
Arch Dis Child
(1984) Intracranial hemorrhage in idiopathic thrombocytopenic purpura
Arch Dis Child
(1994)- et al.
Treatment of chronic childhood immune thrombocytopenic purpura with intramuscular anti-D immunoglobulin
Br J Haematol
(1994)
Cited by (50)
Protocol for the study and treatment of primary immune thrombocytopenia: PTI-2018
2019, Anales de PediatriaThe American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia
2011, BloodCitation Excerpt :In a prospective, randomized trial of 6 cycles of high-dose dexamethasone (0.6 mg/kg/d for 4 days every 4 weeks) and IVIg (800 mg/kg with a second dose if platelet count is less than 30 × 109/L at 48 hours for 6 cycles), complete or partial remissions occurred in 25% (5/20) of patients initially treated with corticosteroids or crossed over to this therapy after failure to respond to IVIg.43 Small prospective observational studies yield similar results with frequent adverse events.44-47 The 1996 ASH guideline noted that numerous agents such as azathioprine, danazol, and interferon have been used in a small number of children and adolescents with chronic or persistent ITP who failed to respond to more conventional therapy.
International consensus report on the investigation and management of primary immune thrombocytopenia
2010, BloodCitation Excerpt :However, side effects such as sleeplessness, aggressive behavior, and loss of concentration are unacceptably high.198 HDMP (given as an oral 7-day course of 30 mg/kg/d for 3 days followed by 20 mg/kg/d for 4 days) has been used as an alternative to IVIg200-202 (evidence level Ib-III). See “First-line/initial treatment options to raise platelet counts in children.”
The cumulative burden of oral corticosteroid side effects and the economic implications of steroid use
2009, Respiratory MedicineCurrent Options for the Treatment of Idiopathic Thrombocytopenic Purpura
2007, Seminars in HematologyCitation Excerpt :In another study, none of the nine patients with chronic ITP responded to high-dose dexamethasone, and five could not tolerate the treatment.41 In children with chronic ITP, few long-lasting remissions have been reported following high-dose dexamethasone42 but in one study, three of seven children achieved a platelet count above 50 × 109/L after 6 months, including one for whom the response was maintained to 1 year.43 In conclusion, corticosteroids remain the cornerstone of treatment for patients with newly diagnosed ITP and should be tried in chronic patients who require treatment.
- ☆
Supported by the Ministero dell' Universitá e della Ricerca Scientifica e Technologica (60% 1995; Dr. Borgna-Pignatti) and by the Associazione Italiana per la Ricerca sul Cancro (Dr. Locatelli).
- ☆☆
Reprint requests: Caterina Borgna-Pignatti, Istituto di Pediatria Universitá di Ferrara, Via Savonarola 9, 44100 Ferrara, Italy.
- ★
0022-3476/97/$5.00 + 0 9/21/77543