Comparison between Pulsed High-Dose Dexamethasone and Daily Corticosteroid Therapy for Adult Primary Immune Thrombocytopenia: A Retrospective Study

Abstract

Objective Recently, pulsed high-dose dexamethasone (HD-Dexa) therapy was proposed as a possible alteration for the classical prednisolone (PSL) therapy for primary immune thrombocytopenia (ITP) patients, however it remains to be confirmed which of these remedies is superior. So the objective of this study is to compare the efficacy and the sustainability of these options. Methods The first-line therapy at our institute for untreated adult ITP cases was accordingly changed as follows, and we retrospectively evaluated the outcomes: 1) daily administration of 0.5-1 mg/kg PSL for 2-4 weeks and subsequently stepwise reduction, 2) one course of HD-Dexa (40 mg/day for four consecutive days, 1xHD-Dexa), 3) three courses of the same dose of HD-Dexa (3xHD-Dexa) repeated biweekly. This study was approved by the ethical committee of the University of Tokyo. Results Twenty-five patients were enrolled consecutively. A good initial response was attained through all the regimens. Meanwhile, time to next treatment for lack of response or relapse was significantly longer in the PSL group than in the other groups (log-rank test, PSL vs. 1xHD-Dexa p<0.001, PSL vs. 3xHD-Dexa p= 0.0053, respectively). Additionally, PSL regimen conferred a significantly longer duration time of response (PSL vs. 1xHD-Dexa p=0.0024, PSL vs. 3xHD-Dexa p=0.028, respectively) and CR (PSL vs. 1xHD-Dexa p=0.012, PSL vs. 3xHD-Dexa p=0.0090, respectively). No patient discontinued the treatment due to side effects in this study. Conclusion PSL regimen was considered to be superior to pulsed HD-Dexa regimens in the sustainability of response.

High-dose dexamethasone vs prednisone for treatment of adult immune thrombocytopenia: a prospective multicenter randomized trial

“…13 Retrospective studies comparing the 2 approaches in smaller cohorts yielded conflicting results [14][15][16] ; thus, RCTs directly comparing these 2 corticosteroid regimens are required to provide higher level evidence of efficacy. The design of this study was based on the standardized definitions and outcome criteria of the IWG.…”

A Randomized Trial of Daily Prednisone versus Pulsed Dexamethasone in Treatment-Naïve Adult Patients with Immune Thrombocytopenia: EIS 2002 Study

“…The discrepancy between the results of our study and the other studies may be reconciled by the accumulating evidence that the efficacy of dexamethasone increases with an increasing number of treatment cycles (table 2). In one retrospective [14] and five prospective studies [10,11,12,13,16,17] a single course of dexamethasone (40 mg, days 1-4) led to initial responses in 62-85% of patients, but long-term remissions were observed in only 8-42%. By contrast, repetitive 28-day dexamethasone cycles were followed by lasting remissions in 59% of patients when treatment was continued one cycle beyond complete remission (median 2.5, maximum 6 cycles) [9].…”

“…Lasting responses were frequent in the initial report [3], but subsequent studies were unable to confirm this finding [4,5,6,7,8,9]. Modifications of the regimen have also been used as a first-line approach [9,10,11,12,13,14,15,16,17]. In 2002, we embarked on a prospective, open-label, randomized, multicenter trial comparing standard prednisone with six cycles of pulsed dexamethasone in treatment-naïve adults with ITP (Essen ITP Study, EIS 2002).…”

Safety and efficacy of eltrombopag plus pulsed dexamethasone as first‐line therapy for immune thrombocytopenia

“…A review of nine prospective and retrospective studies involving a total of 589 newly diagnosed ITP subjects (Appendix 1) (Cheng et al , ; Bae et al , ; Nakazaki et al , ; Gudbrandsdottir et al , ; Li et al , ; Din et al , ; Sun et al , ; Wei et al , ; Wang et al , ) showed that the sustained response rates defined as platelet counts >30–50 × 10 9 /l for 3–12 months without any ITP therapy after one to three courses of pulsed dexamethasone were in the range of 8–42%, with a mean of 33% (95% CI 29–36%). In some of the studies, pulsed dexamethasone was given at intervals of 7–14 days, counting only those studies that administered either only one single course of pulsed dexamethasone or up to three courses at four‐week intervals (Cheng et al , ; Bae et al , ; Nakazaki et al , ; Li et al , ; Sun et al , ). The mean sustained response rate of these five studies was 31% (95% CI 25–36%).…”

“…Cheng et al () previously reported that a single course of pulsed dexamethasone 40 mg daily for four days resulted in an 85% initial response rate and 42% (CI 33–51%) of the subjects maintained sustained platelet counts >50 × 10 9 /l at six‐month follow‐up. We reviewed nine prospective and retrospective studies evaluating the long‐term efficacy of pulsed high‐dose dexamethasone as first‐line treatment for newly diagnosed adult primary ITP (Cheng et al , ; Bae et al , ; Nakazaki et al , ; Gudbrandsdottir et al , ; Li et al , ; Din et al , ; Sun et al , ; Wei et al , ; Wang et al , ). Some of these studies defined sustained response as platelet counts greater than 30 × 10 9 /l at 3–12 months off therapy (Bae et al , ; Nakazaki et al , ; Li et al , ; Din et al , ; Sun et al , ; Wei et al , ; Wang et al , ), while others used a platelet threshold of greater than 50 × 10 9 /l at six months (Cheng et al , ; Gudbrandsdottir et al , ).…”