scholarly journals A second-line escalating treatment strategy for children with severe chronic immune thrombocytopenia: A retrospective data from a single-center

2020 ◽  
Author(s):  
lingling FU ◽  
Jie Ma ◽  
Hao Gu ◽  
Jingyao Ma ◽  
Yunyun Wei ◽  
...  
Keyword(s):  
Relapse Rate ◽  
Treatment Strategy ◽  
Immune Thrombocytopenia ◽  
Low Dose ◽  
High Dose ◽  
Second Line ◽  
Single Center ◽  
High Dose Dexamethasone ◽  
Number Of Patients ◽  
Chronic Immune Thrombocytopenia

Objective: To analyze the effect of a novel second-line escalating treatment strategy (high-dose dexamethasone (HDD), low-dose rituximab to eltrombopag) for children with severe chronic immune thrombocytopenia (SCITP). Materials and Methods: This study was a single-center, retrospective cohort study. The second-line escalating strategy included 3 steps: Step I (6 courses high-dose dexamethasone: HDD), Step II (HDD combined with low-dose rituximab), and Step III (eltrombopag). Results: A total of 30 cases (18 males and 12 females) were included; the median age was 8.83 (1.42-13.9) year-old, the duration time of ITP was 20.5 (12.0-96.0) months, and the platelet counts were 15 (3-29) ×109/L. After the median 14 (12-37) months’ treatment, the remission rate was 36.7% (11/30), and the sustained response (SR) rate was 68.2% (15/22). In eltrombopag (step III) cases, 47.5% (8/17) maintained platelet ≥50×109/L, 37.5% (3/8) dose tapering, and 25% (2/8) were successfully discontinued from medication. The number of patients at 12th, 24th, and 36th months was 30, 7, and 2, with the total response (TR) and remission rates of 80% (36.7%), 57.1% (28.6%), and 50% (50%), respectively. The total relapse rate was 26.7% (8/30)，three cases(75%, 3/4)from Step II and 5 cases (41.7% ,5/12)from Step III, none in Step I. Conclusion: The new second-line escalating strategy for children SCITP has an effective improving rate with 36.7% remission and 68.2% SR; 30% could benefit and retain stable response from HDD treatment. Combined treatment with eltrombopag can reduce the relapse rate of low-dose rituximab.

High response rate to low-dose rituximab plus high-dose dexamethasone as frontline therapy in adult patients with primary immune thrombocytopenia

2013 ◽  
Vol 90 (6) ◽  
pp. 494-500 ◽  
Author(s):  
David Gómez-Almaguer ◽  
Luz Tarín-Arzaga ◽  
Brizio Moreno-Jaime ◽  
José Carlos Jaime-Pérez ◽  
Adrián Alejandro Ceballos-López ◽  
...  
Keyword(s):  
Immune Thrombocytopenia ◽  
Low Dose ◽  
Response Rate ◽  
High Response Rate ◽  
High Response ◽  
Adult Patients ◽  
High Dose ◽  
High Dose Dexamethasone ◽  
Primary Immune Thrombocytopenia ◽  
Frontline Therapy

Long-Term Effect of High-Dose Dexamethasone with or without Low-Dose Dexamethasone Maintenance in Untreated Immune Thrombocytopenia

2014 ◽  
Vol 133 (1) ◽  
pp. 124-128 ◽  
Author(s):  
Banhe Din ◽  
Xingwen Wang ◽  
Yuye Shi ◽  
Yufeng Li
Keyword(s):  
Immune Thrombocytopenia ◽  
Low Dose ◽  
Overall Response Rate ◽  
Control Group ◽  
High Dose ◽  
High Dose Dexamethasone ◽  
Group 4 ◽  
Long Term Effect ◽  
Group 2

Background: To tackle the problems associated with high-dose dexamethasone (HD-DXM) in patients with immune thrombocytopenia (ITP). Aim: To compare the efficacy of HD-DXM with or without low-dose dexamethasone maintenance in untreated ITP patients. Results: Dexamethasone (40 mg/day) was given in 4-day pulses every 14 days for 3 cycles in 61 patients with ITP. Among them, 30 cases were given dexamethasone (0.035 mg/kg per day) for maintenance between pulsed HD-DXM and after 3 HD-DXM courses (HD-DXM-M group) and another 31 cases did not receive dexamethasone maintenance (HD-DXM-nM group). The control group comprised the patients who received prednisone (prednisone group). The following results were obtained: (1) at the end of the 3rd cycle, the overall response rate (ORR) was higher in the HD-DXM group than in the prednisone group; (2) the ORR of the HD-DXM group peaked after the 3rd cycle; (3) the ORR after each course was higher in the HD-DXM-M group than in the HD-DXM-nM group; (4) in the 12th month after HD-DXM discontinuation, the relapse rate of the HD-DXM-M group was lower than that of the other groups (prednisone and HD-DXM-nM). Conclusion: Treatment with 3 cycles of HD-DXM pulses with low-dose dexamethasone maintenance is an effective method for untreated ITP.

scholarly journals Romiplostim, Low-Dose Rituximab and High-Dose Dexamethasone Combination in Newly Diagnosed Immune Thrombocytopenia: Another "Total Therapy" Pilot Study

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 1014-1014
Author(s):  
Perla R. R. Colunga-Pedraza ◽  
Mónica Bustillos Muñoz ◽  
Fernando De La Garza ◽  
Andres Gomez-De Leon ◽  
Edgar Ulises Coronado-Alejandro ◽  
...  
Keyword(s):  
Immune Thrombocytopenia ◽  
Low Dose ◽  
Initial Response ◽  
High Dose ◽  
Newly Diagnosed ◽  
Front Line ◽  
High Dose Dexamethasone ◽  
Line Therapy ◽  
Total Therapy

Abstract Background: Primary immune thrombocytopenia (ITP) is an acquired autoimmune disorder that results from accelerating platelet clearance, destruction, and production. Front-line therapy for newly diagnosed ITP includes corticosteroids, intravenous immune globulin, or anti-D immunoglobulin. However, after these single-agent therapies, relapses will occur in half of patients. We previously reported the safety, feasibility, and efficacy of the combination of dexamethasone, low-dose rituximab, and the thrombopoietin receptor agonist (TPO-Ra) eltrombopag as front-line treatment in newly diagnosed ITP. Romiplostim, another TPO-Ra is approved by the FDA for patients with chronic ITP. However, the safety, tolerability, and efficacy of romiplostim combined with low-dose rituximab, and high-dose dexamethasone in newly diagnosed ITP remain unknown. Objective: Our primary objectives were safety, and tolerability. Secondary objectives were initial response and relapse incidence. Methods: An open-label, single-arm study was performed in Hospital Universitario "Dr. José Eleuterio González" in Monterrey, Mexico (Clinical trials.gov NCT04588194). Eligible patients were newly diagnosed ITP patients, treatment-naïve, ≥18 years, with a platelet count ≤30×10 9/L. Patients were excluded if they had an active infection, pregnancy, or malignant disease. The treatment regimen was romiplostim (2 mcg/kg weekly, four doses), low-dose rituximab (100 mg weekly, four doses) and high-dose dexamethasone (40 mg PO, days 1-4). Dexamethasone was allowed up to 3 cycles. Partial (PR) and complete responses (CR) were defined as an increase in platelet counts ≥30×10 9/L (at least a doubling of the baseline count) and ≥100×10 9/L, respectively. Results: We included ten consecutive patients. The median age was 34.5 years (range, 17-63). Seven patients were men (70%). The median platelet account at diagnosis was 6x10 9/L (range 0-23), and median follow-up was 180 days (range 30-270). The median number of romiplostim doses was 3.5 (range 1-4), and three (30%) patients required dose adjustment due to thrombocytosis. All but one patient achieved response (CR or PR) at a median of 7 days (range 7-28). Five patients (50%) achieved CR at 28 days of treatment, and four patients (40%) PR. No significant adverse effects have occurred during treatment; one patient presented grade 1 myalgia and the other a grade 2 soft tissue infection. Five patients (50%) relapsed during follow-up. Recently, four (40%) patients remain in CR and three (30%) in PR. Conclusion: The combination of romiplostim, low-dose rituximab, and high-dose dexamethasone was safe and effective. This "total therapy" approach was associated with minimum side effects and rapid initial response. Prospective validation in a larger sample is needed. Figure 1 Figure 1. Disclosures Gomez-Almaguer: Janssen: Honoraria, Speakers Bureau; Roche: Honoraria, Speakers Bureau; Bristol-Myers-Squibb: Honoraria, Speakers Bureau; Takeda: Honoraria, Speakers Bureau. OffLabel Disclosure: Romiplostim in firs-line therapy for immune thrombocytopenia

Low-Dose Rituximab and High-Dose Dexamethasone As Front-Line Therapy in Adult Patients with Primary Immune Thrombocytopenia

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 3332-3332
Author(s):  
David Gomez-Almaguer ◽  
Luz C. Tarin-Arzaga ◽  
Brizio Moreno-Jaime ◽  
Jose C. Jaime-Pérez ◽  
Adrián A. Ceballos-López ◽  
...  
Keyword(s):  
Platelet Count ◽  
Immune Thrombocytopenia ◽  
Low Dose ◽  
Complete Response ◽  
High Dose ◽  
Newly Diagnosed ◽  
Front Line ◽  
High Dose Dexamethasone ◽  
Line Therapy ◽  
Primary Immune Thrombocytopenia

Abstract Abstract 3332 Introduction Corticosteroids are the initial standard therapy for primary immune thrombocytopenia (ITP). A short course of high-dose dexamethasone as initial therapy is an alternative to prednisone with high initial responses, but about half of the patients relapse. Low dose rituximab has been used in the treatment of relapsed ITP, showing a similar activity to the standard dose. The aim of this prospective study was to evaluate the efficacy, safety and response duration of low-dose rituximab plus short pulses of high-dose dexamethasone as front-line therapy in newly diagnosed ITP adults. Methods Patients ≥18 years old with newly diagnosed received dexamethasone, 40 mg/day/i.v. for 4 consecutive days (+1, +2, +3, +4), rituximab was administered at a fixed dose of 100 mg as an i.v. infusion weekly for 4 consecutive doses (days +1, +8+, +15 and +22). The use of rescue therapy was allowed, using the same schedule of dexamethasone, before day 30, but only if the platelet count was < 20×109/L. A complete blood cell count was performed at enrollment, weekly for the first 28 days, monthly until month 6 and then every 3 months. The degree of response was defined as follows: a complete response (CR) was a platelet count ≥ 100 × 109/L; a complete sustained response (CSR) was considered if it was maintained for six months. Partial response (PR) was defined as a platelet level between 50 and 100 × 109/L; an overall response (OR) was defined as partial or complete response. Patients with a platelet count <30 × 109/L were considered non-responders (NR). Time-to-response (TTR) and time-to-complete response (TCR) were also assessed, as well as the safety profile and side effects incidence according to the National Cancer Institute Common Toxicity Criteria version 3.0. Results Twenty-one consecutive patients were enrolled from December 2009 to December 2011 (17 women and 4 men); median age was 51 years (range, 18–82). The median platelet count at baseline was 5.19 × 109/L (range, 0.3–24.2 × 109/L). Patients were followed for a median of 12 months (range, 1–25). At day +28, sixteen patients (76.2%) had CR, three patients had PR (14.3%), with the OR being 90.5%. At month six, 16 of 21 patients (76.2%) had achieved CSR. Seven patients received a second course of dexamethasone (one at day +8 and six at day+15) (Table 1). The median duration of response was 12 months (range, 7–25 months). The median time to reach TTR and TCR was 8 days (range, 4–28). Partial response was achieved in two patients who were further treated with danazol and prednisone, being subsequently splenectomized. The relapse rate was 15.8%. The 6- and 12- month cumulative RFS were both 84%; the 6- and 12-month cumulative TFS probabilities were 94% and 87%, respectively (Fig. 1). Overall, combination therapy in our group was well tolerated with a lower incidence of adverse effects during infusion in this trial according to the data reported in previous studies. Conclusions The combination of low-dose rituximab and high-dose dexamethasone as front-line therapy for adults with ITP was effective and safe with a high OR rate and a low incidence of relapse. These data need to be confirmed in a prospective randomized clinical trial including a sample size with enough power to reach statistically significant conclusions. Disclosures: No relevant conflicts of interest to declare.

scholarly journals A Prospective Study to Evaluate the Efficacy, Safety and Response Duration of Newly Diagnosed Adult Immune Thrombocytopenia Patients Treated with Low Dose Rituximab and High Dose of Dexamethasone

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 4921-4921 ◽  
Author(s):  
Tuphan Kanti Dolai ◽  
Soumya Mukherjee ◽  
Prakas Kumar Mandal ◽  
Rajib De ◽  
Prantar Chakrabarti
Keyword(s):  
Prospective Study ◽  
Immune Thrombocytopenia ◽  
Low Dose ◽  
Conflicts Of Interest ◽  
Response Duration ◽  
Sustained Response ◽  
Fixed Dose ◽  
High Dose ◽  
High Dose Dexamethasone ◽  
Line Therapy

Abstract Introduction Primary immune thrombocytopenia (ITP) is an acquired autoimmune disorder that involves antibody and cell mediated destruction of platelets as well as suppression of their production. Corticosteroids are initial standard therapy in adults. Initially Rituximab (dose375mg/m2) was approved as one of the important second line therapy in chronic ITP. In the present prospective study, we will evaluate the efficacy, safety and response duration of low dose Rituximab (100mg) and high dose Dexamethasone as a front line therapy in new onset adult ITP cases. Methods Inj. Rituximab at a fixed dose of 100mg intravenous infusion for four doses (day 1,8,15,22) and Dexamethasone tablet 40 mg/daily PO for four days in a fifteen days' interval (day 1-4 and day 15-18). Response rate evaluated according to published guidelines. Results Total no. of cases was 15. Male 5, female 10. Age range 18 to 62 years with median age of 36 years. Course completed in 15 patients. Median follow up of the patients was 6 months. Complete response (CR) was achieved after 1st dose of rituximab in 3 cases, 2 after 2nd dose and 4 after 4th dose of Rituximab. Partial response (PR) was achieved in 2 cases after completion of therapy and no response seen in 4 cases. Seven cases (46.66%) achieving CR maintaining sustained response after 6 months of completion of therapy. Two cases who achieved CR initially reverted to PR after 6 months with dropping of platelet count but without any evidence of bleeding symptoms. Minor side effects like GI intolerance, nausea, vomiting occurred in 3 patients, High blood sugar level during treatment seen in 2 patients and severe adverse effects like pneumonitis and intestinal obstruction seen in 2 patients who needed hospitalization. We did not observe any infusion related reaction. Conclusions CR and PR rate was 60% (9/15) and 14%(2/15) respectively. Six months after completion of therapy 77.8% (7/9) showed sustained response after achieving CR. Low dose Rituximab and high dose Dexamethasone is a useful treatment option in adult acute ITP patients. Disclosures No relevant conflicts of interest to declare.

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