scholarly journals Immunomodulatory and immunosuppressive drug protocols in the treatment of canine primary immune thrombocytopenia, a scoping review

2021 ◽  
Vol 63 (1) ◽  
Author(s):  
Peter Spanner Kristiansen ◽  
Lise Nikolic Nielsen
Keyword(s):  
Adverse Events ◽  
Outcome Measures ◽  
Scoping Review ◽  
Recovery Time ◽  
Immune Thrombocytopenia ◽  
Immunosuppressive Drugs ◽  
Immunosuppressive Drug ◽  
Controlled Trials ◽  
Platelet Recovery ◽  
Primary Immune Thrombocytopenia

AbstractPrimary immune thrombocytopenia (ITP) is a cause of severe thrombocytopenia in dogs. Immunosuppressive corticosteroid drugs are frequently used in the management of ITP, but treatment failure may occur. Immunomodulatory and non-corticosteroid immunosuppressive drugs might improve outcomes from therapy either alone or in combination with corticosteroids. The objectives of this scoping review were (1) to evaluate the current evidence relating to immunomodulatory and immunosuppressive drug protocols in the treatment of canine ITP, and (2) to answer the clinical question, whether or not therapy with immunomodulatory or non-corticosteroid immunosuppressive drugs alone or in combination with corticosteroids could improve outcome, compared to therapy with corticosteroids alone. A literature search was performed in the electronic databases of Agricola, CAB Abstracts, Embase, Medline and Web of Science for publications in November 2019 and again February 1, 2021. Selection criteria were relatively strict and included peer-reviewed research papers reporting outcome measures from immunomodulatory and immunosuppressive drug protocols in the treatment of canine ITP with a pre-therapeutic mean or median platelet count < 50,000/µL as a strict criterion for inclusion. Studies were evaluated if they had an appropriate diagnostic work up to exclude underlying conditions. Outcome measures and adverse events were compared between drug protocols both within studies and between studies. The search identified 456 studies, with six studies being eligible for inclusion. The studies were mostly case series while two were randomized controlled trials. Level of evidence varied with an overall uncertain subject enrollment, small groups, inadequate description and variable use of drug protocols or outcome measures. For outcomes such as platelet recovery time and duration of hospitalization, an improvement was observed using adjunctive therapy (human intravenous immunoglobulin) compared to therapy with corticosteroids alone. For outcomes of complete platelet recovery time, survival (6-month), mortality and relapse, no improvement was observed using adjunctive drugs compared to corticosteroids alone. Specifically, therapy with mycophenolate mofetil alone and adjunctive azathioprine were associated with more severe adverse events compared to other drug protocols. Evidence relating to immunomodulatory and immunosuppressive drug protocols in the treatment of canine ITP was of variable quality. Future larger case-controlled trials are required for determination of optimal treatment protocols in canine ITP.

scholarly journals Efficacy and Safety of the Combination Treatment of Rituximab and Dexamethasone for Adults with Primary Immune Thrombocytopenia (ITP): A Meta-Analysis

2018 ◽  
Vol 2018 ◽  
pp. 1-12 ◽  
Author(s):  
Jia Wang ◽  
Ya Li ◽  
Chong Wang ◽  
Yayue Zhang ◽  
Chong Gao ◽  
...  
Keyword(s):  
Randomized Controlled Trials ◽  
Combination Treatment ◽  
Immune Thrombocytopenia ◽  
Response Rate ◽  
Meta Analysis ◽  
Controlled Trials ◽  
Efficacy And Safety ◽  
Randomized Controlled ◽  
Significant Difference ◽  
Primary Immune Thrombocytopenia

Objective.To conduct a meta-analysis, assessing the efficacy and safety of the combination treatment of dexamethasone and rituximab for adults with ITP (primary immune thrombocytopenia).Methods.Randomized controlled trials that compared rituximab and dexamethasone combination treatment to dexamethasone monotherapy in the treatment of adults with ITP were collected by searching Pubmed, Embase, Cochrane, China National Knowledge (CNKI), Wanfang database, and Sino Med. We conducted pooled analyses on OR (overall response) rate, CR (complete response) rate, PR (partial response) rate, SR (sustained response) rate, R (relapse) rate, change in Treg cell count (mean [SD]), and AE (adverse event). GRADE pro scale was used to assess the quality of the evidence. Publication bias was assessed with Egger’s test method.Results.A total of 11 randomized controlled trials were eligible for inclusion. The overall efficacy estimates favored combination arm in terms of OR rate at month 3, CR rate at week 4 and month 3, SR rate, and Treg cell count at week 2. Subgroup analysis showed that females obtained a higher OR rate than males did at week 4. No significant difference was found in pooled analysis of relapse rate between combination arm and monotherapy arm. The comparison of serious AE and other AEs showed no significant difference either. A total of 19 outcomes were assessed by GRADE pro software, of which 79% (15/19) was scaled as moderate-to-high level. Publication bias existed in studies on OR at week 4 (P=0.025), CR at week 4 (P=0.017), infection (P=0.006), and rash (P=0.028) of the AEs.Conclusion.Dexamethasone combined with rituximab can provide a better long-term response in the treatment of adults with ITP and will not increase the risk of adverse effects.

Dexamethasone plus rituximab yields higher sustained response rates than dexamethasone monotherapy in adults with primary immune thrombocytopenia

Blood ◽  
2010 ◽  
Vol 115 (14) ◽  
pp. 2755-2762 ◽  
Author(s):  
Francesco Zaja ◽  
Michele Baccarani ◽  
Patrizio Mazza ◽  
Monica Bocchia ◽  
Luigi Gugliotta ◽  
...  
Keyword(s):  
Platelet Count ◽  
Adverse Events ◽  
Salvage Therapy ◽  
Immune Thrombocytopenia ◽  
Sustained Response ◽  
Effective Treatment Option ◽  
Serious Adverse Events ◽  
Primary Immune Thrombocytopenia ◽  
Grade 3 ◽  
To Receive

Abstract Previous observational studies suggest that rituximab may be useful in the treatment of primary immune thrombocytopenia (ITP). This randomized trial investigated rituximab efficacy in previously untreated adult ITP patients with a platelet count of 20 × 109/L or less. One hundred three patients were randomly assigned to receive 40 mg/d dexamethasone for 4 days with or without 375 mg/m2 rituximab weekly for 4 weeks. Patients who were refractory to dexamethasone alone received salvage therapy with dexamethasone plus rituximab. Sustained response (ie, platelet count ≥ 50 × 109/L at month 6 after treatment initiation), evaluable in 101 patients, was greater in patients treated with dexamethasone plus rituximab (n = 49) than in those treated with dexamethasone alone (n = 52; 63% vs 36%, P = .004, 95% confidence interval [95% CI], 0.079-0.455). Patients in the experimental arm showed increased incidences of grade 3 to 4 adverse events (10% vs 2%, P = .082, 95% CI, −0.010 to 0.175), but incidences of serious adverse events were similar in both arms (6% vs 2%, P = .284, 95% CI, −0.035 to 0.119). Dexamethasone plus rituximab was an effective salvage therapy in 56% of patients refractory to dexamethasone. The combination of dexamethasone and rituximab improved platelet counts compared with dexamethasone alone. Thus, combination therapy may represent an effective treatment option before splenectomy. This study is registered at http://clinicaltrials.gov as NCT00770562.

Recombinant Human Thrombopoietin and Rituximab vs Rituximab Monotherapy in Corticosteroid-Resistant Primary Immune Thrombocytopenia: a Multicenter Randomized Controlled Study

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 329-329
Author(s):  
Miao Xu ◽  
Ping Qin ◽  
Xiaoyuan Dong ◽  
Jie Li ◽  
Haiyan Zhang ◽  
...  
Keyword(s):  
Adverse Effects ◽  
Adverse Events ◽  
Immune Thrombocytopenia ◽  
Wilcoxon Test ◽  
Fixed Dose ◽  
Combination Group ◽  
Monotherapy Group ◽  
Platelet Counts ◽  
Primary Immune Thrombocytopenia ◽  
Time To Relapse

Abstract Background Though great progresses had been achieved in the management of primary immune thrombocytopenia (ITP), still 25%-30% of the patients show no response to the conventional therapy. Both thrombopoietin (TPO) and rituximab (RTX) are recommended as the second-line treatments. TPO has shown potent activity in ITP, but the therapeutic efficacy is dependent on continual administration. Rituximab is an anti-CD20 chimeric monoclonal antibody which could induce prolonged remission in ITP. However, longer time to response was also reported. The combination of rhTPO and rituximab could complement each other in both mechanism of action and time window, exert powerful effect, which may be a choice for the glucocorticosteroids-resistant/relapsed patients. Method Patients with platelet counts ≤ 30 x109/L, or ≤ 50 x109/L with bleeding symptoms from 12 centers were enrolled in the study. Subjects enrolled in the study were assigned randomly as 1: 2 to RTX group and rhTPO plus RTX group. Eligibility criteria were set according to the recently published guideline (Rodeghiero F, et al, Blood 2009). Approval for the study was obtained from the Ethics Committee of the School of Medicine and Qilu Hospital. Informed consent in accordance with the Declaration of Helsinki was given to each patient. Rituximab (MabThera; Roche) was given with a fixed dose of 100mg weekly for four weeks. And in the combination group, rhTPO (Tebiao, 3SBio, China) was added in the regimen with a dose of 300U/kg/day in the first 14 days, and a modified frequency of rhTPO was given according to the platelet counts after two weeks, but no longer than four weeks. Primary outcomes include response (R), complete response (CR), no response (NR) and relapse. All the criteria were consent with the definition and outcome criteria. (Rodeghiero F, et al. Blood, 2009.) Secondary outcomes are listed below. Time to response (TTR) was considered as the duration from baseline to response. Time to relapse was also measured with Kaplan-Meier analysis. Besides, safety was reflected as adverse events graded according to the Common Toxicity Criteria. This clinical trial was registered at http://clinicaltrials.gov as NCT 01525836. Result A total of 114 patients were recruited: monotherapy group (n = 35) and combination group (n = 79). All patients failed to response to glucocorticosteroids or relapsed after corticosteroids treatment. Response rate was reached in 71.4% (25/35) of patients in monotherapy group vs. 78.5% (62/79) in the RTX and rhTPO combination group, while complete responses were achieved as 10/35 (28.6%) in RTX monotherapy group and 34/79 (43.0%) in rhTPO plus RTX group respectively. Median time to response was 28 days (range 4-90 days) in RTX group, while in RhTPO plus RTX group, TTR was 7 days (range 4-28 days) (P = 0.002). There was significantly longer time to relapse in the combination group (shown in Fig. 1). Adverse effects were observed in RTX group in 5 patients, most of whom were affected with inflammation. One patient was reported as viral myocarditis, and there were also complaints about chill, rash and hyperpyrexia. And incidence of adverse events was slightly increased in rhTPO plus RTX group. There were 14 adverse effects reported in the combination group, such as fatigue, pulmonary inflammatory, but all were below Grade 2. Conclusion Our findings suggest that combination of RTX and RTX in ITP yields shorter time to response compared with monotherapy of RTX, and moreover, combination extended time to relapse. Thus, combination therapy may represent an effective treatment option for glucocorticosteroids-resistant or relapsed patients. The results were compared with Gehan-Breslow-Wilcoxon Test. (P = 0.0140) Disclosures: No relevant conflicts of interest to declare.

Physical performance outcome measures used in exercise interventions for adults with childhood-onset disabilities: A scoping review

2020 ◽  
Vol 47 (4) ◽  
pp. 359-380
Author(s):  
Jessica Z. Song ◽  
Margot Catizzone ◽  
Kelly P. Arbour-Nicitopoulos ◽  
Dorothy Luong ◽  
Laure Perrier ◽  
...  
Keyword(s):  
Cerebral Palsy ◽  
Physical Performance ◽  
Outcome Measures ◽  
Scoping Review ◽  
Brain Injuries ◽  
Controlled Trials ◽  
Cochrane Central Register ◽  
Childhood Onset ◽  
Exercise Interventions ◽  
Performance Outcome

BACKGROUND: People with childhood-onset disabilities face unique physical and social challenges in adulthood. Exercise interventions may improve physical performance in children, but there is a lack of research on adults. OBJECTIVE: To describe studies that investigate exercise interventions and to evaluate the quality of physical performance outcome measures for adults with childhood-onset disabilities. METHODS: Eligible studies reported on exercise interventions for adults (ages 16+) with cerebral palsy, spina bifida, or acquired brain injuries. Only randomized controlled trials published in English from 2008 to 2019 were included. MEDLINE, CINAHL, PEDro, EMBASE, and Cochrane Central Register of Controlled Trials were searched. Two reviewers independently screened studies and abstracted data. RESULTS: This scoping review included 4 trials reporting on cerebral palsy only. Three strength training programs found significant improvements in gait, and one mixed training program found significant improvements in strength and fitness. Only two outcome measures used are valid/reliable for adults (6 Minute Walk Test and Borg-20 Grades). CONCLUSION: Certain interventions may improve physical performance, but there is a lack of research on appropriate exercise interventions and physical performance outcome measures for adults with childhood-onset disabilities. Different exercise interventions should be investigated using larger sample sizes and outcome measures should be standardized.

Early versus Late Initiation of Renal Replacement Therapy: Have We Reached the Consensus? An Updated Meta-Analysis

Nephron ◽  
2021 ◽  
pp. 1-15
Author(s):  
Girish C. Bhatt ◽  
Rashmi Ranjan Das ◽  
Amit Satapathy
Keyword(s):  
Renal Replacement Therapy ◽  
Adverse Events ◽  
Replacement Therapy ◽  
Outcome Measures ◽  
Data Extraction ◽  
Secondary Outcome ◽  
Controlled Trials ◽  
Cochrane Central Register ◽  
Study Selection ◽  
Late Initiation

<b><i>Objectives:</i></b> The objective of this study is to compare early versus late/standard initiation of renal replacement therapy (RRT) in patients with acute kidney injury (AKI). <b><i>Data Sources:</i></b> MEDLINE/PubMed, Embase, Google Scholar, Cochrane Central Register of Controlled Trials, and the Cochrane renal group till August 15, 2020. <b><i>Study Selection:</i></b> Randomized controlled trials (RCTs) comparing early versus late initiation of RRT in patients with AKI were included. The primary outcome measures were all-cause mortality and dialysis dependence on day 90. Secondary outcome measures were length of stay, recovery of renal functions, and adverse events. <b><i>Data Extraction:</i></b> Two authors independently performed study selection and data extraction using data extraction forms. <b><i>Data Synthesis:</i></b> A total of 14 RCTs with 5,234 participants were included. Three trials had low risk of bias in all the domains. There was no significant difference in the overall mortality (risk ratio (RR): 0.99; 95% confidence interval (CI): 0.89, 1.10; moderate certainty of evidence), day 30 mortality (RR: 1.0; 95% CI: 0.91, 1.09; high certainty of evidence), day 90 mortality (RR: 1.00; 95% CI: 0.88, 1.13; high certainty of evidence), and ICU mortality (RR: 1.00; 95% CI: 0.90, 1.10; moderate certainty of evidence) between the early versus late RRT. Dialysis dependence on day 90 was significantly higher in the patients assigned to early RRT (RR: 1.55; 95% CI: 1.15, 2.09; moderate certainty of evidence). The treatment-emergent adverse events (hypophosphatemia and hypotension) were significantly higher in the patients assigned to early RRT. <b><i>Conclusion:</i></b> There is no added benefit of early initiation of RRT in patient with AKI; this may lead to treatment-emergent adverse events. Delaying the initiation of RRT with close monitoring and initiating RRT for emergent indications should be the acceptable criterion in critical care nephrology. <b><i>Prospero Registration:</i></b> CRD42016043092.

scholarly journals Eye acupuncture for pain conditions: a scoping review of clinical studies

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Yuan Chi ◽  
Jürgen Barth ◽  
Mei Wang ◽  
Nicola Robinson ◽  
Zan-Hua Li ◽  
...  
Keyword(s):  
Adverse Events ◽  
Scoping Review ◽  
Clinical Studies ◽  
Lumbar Disc ◽  
Case Series ◽  
Lumbar Pain ◽  
Cochrane Library ◽  
Controlled Trials ◽  
Beneficial Effects ◽  
Eye Acupuncture

Abstract Background Chinese eye acupuncture, focuses on treating different diseases by applying needle stimulation around the orbit of the eye. Since 1970, it has been used in China for the management of pain-related disorders. This scoping review systematically collected clinical studies on the use of eye acupuncture to treat pain conditions and identify any adverse events. Methods Six databases including PubMed, the Cochrane Library, CNKI, VIP, Wan Fang Data and SinoMed were searched from 1970 to March 2019. Randomized controlled trials (RCTs), clinical controlled trials (CCTs) and case series on eye-acupuncture for pain conditions meeting the inclusion criteria were identified. Data were extracted on patients, interventions, details of eye acupuncture, control treatments and outcomes. Results Searches identified 81 clinical studies and a trend demonstrating an increasing number of published studies. All studies were conducted in China and published in Chinese. These included, 45 (55.6%) RCTs, 5 (6.2%) CCTs, and 31 (38.3%) case series, treating 7113 patients with 44 different pain-related diseases or symptoms. The most frequently reported conditions were headache (18, 16.2%), acute lumbar pain (7, 6.3%) and lumbar disc herniation (7, 6.3%). Treatment using small needles (φ0.25 × 13 mm), retained ≤30 min, needling the horizontal outer orbital edge and the avoidance of manipulation during treatment were the most frequent descriptions of the interventions used. Eye acupuncture was used alone in about half of the studies and of the remaining studies it was combined with other treatment. All studies suggested some beneficial effects including: pain relief, improved quality of life and mental health, and 18 (22.2%) adverse events. Conclusion Eye acupuncture, predominantly studied in China, may be a promising intervention for managing diverse pain conditions. However, given the variety of study designs and reported treatment outcomes, conclusions about the evidence for eye acupuncture for specific conditions are not possible at this stage.

scholarly journals Outcomes tested in non-pharmacological interventions in mild cognitive impairment and mild dementia: a scoping review

BMJ Open ◽  
2020 ◽  
Vol 10 (4) ◽  
pp. e035980 ◽  
Author(s):  
Elyse Couch ◽  
Vanessa Lawrence ◽  
Melissa Co ◽  
Matthew Prina
Keyword(s):  
Cognitive Impairment ◽  
Mild Cognitive Impairment ◽  
Outcome Measures ◽  
Scoping Review ◽  
Controlled Trials ◽  
Pharmacological Treatments ◽  
Pharmacological Interventions ◽  
Pilot Studies ◽  
Mild Dementia ◽  
Wide Range

ObjectivesNon-pharmacological treatments are an important aspect of dementia care. A wide range of interventions have been trialled for mild dementia and mild cognitive impairment (MCI). However, the variety of outcome measures used in these trials makes it difficult to make meaningful comparisons. The objective of this study is to map trends in which outcome measures are used in trials of non-pharmacological treatments in MCI and mild dementia.DesignScoping review.Data sourcesEMBASE, PsychINFO, Medline and the Cochrane Register of Controlled Trials were searched from inception until February 2018. An additional search was conducted in April 2019EligibilityWe included randomised controlled trials (RCTs) testing non-pharmacological interventions for people diagnosed with MCI or mild dementia. Studies were restricted to full RCTs; observational, feasibility and pilot studies were not included.Charting methodsAll outcome measures used by included studies were extracted and grouped thematically. Trends in the types of outcome measures used were explored by type of intervention, country and year of publication.Results91 studies were included in this review. We extracted 358 individual outcome measures, of which 78 (22%) were used more than once. Cognitive measures were the most frequently used, with the Mini-Mental State Examination being the most popular.ConclusionsOur findings highlight an inconsistency in the use of outcome measures. Cognition has been prioritised over other domains, despite previous research highlighting the importance of quality of life and caregiver measures. To ensure a robust evidence base, more research is needed to highlight which outcome measures should be used over others.PROSPERO registration numberCRD42018102649.

P8: CHEMOTHERAPY INDUCED IMMUNE THROMBOCYTOPENIA-AN ENTITY TO KEEP IN MIND!

2016 ◽  
Vol 64 (3) ◽  
pp. 820.2-820
Author(s):  
P Draksharam ◽  
J Park ◽  
G Sidhu
Keyword(s):  
Flow Cytometry ◽  
Platelet Count ◽  
Recovery Time ◽  
Immune Thrombocytopenia ◽  
Chemotherapeutic Agents ◽  
Metastatic Colon Cancer ◽  
Platelet Recovery ◽  
Platelet Antibodies ◽  
Drug Dependent ◽  
Platelet Transfusions

Purpose of StudyThrombocytopenia during chemotherapy is not always due to myelosuppression. We report an unusual case of isolated acute thrombocytopenia after oxaliplatin and irinotecan administration. We reviewed 11 reported cases to better understand the nature of the presentation and variability in response to treatment.Case ReportPatient is a 63 year old female with metastatic colon cancer treated with palliative chemotherapy with FOLFOX. Follwing her 14th cycle she had an episode of acute drop in platelet count to 8,000/microliter. Peripheral smear revealed no evidence of thrombotic microangiopathy. She was managed with supportive platelet transfusions with slow recovery of platelet count. Subsequently she was treated with second line chemotherapy with FOLFIRI. Following the first cycle of Irinotecan, she again had a catastrophic drop in platelets from 136,000/microliter to 6,000/microliter within 10 hours. Due to this recurrent episode, a drug mediated thrombocytopenia was suspected and work up was initiated. She was initially treated with dexamethasone without a significant response. Platelet count normalized after 7 days with supportive platelet transfusions.Methods UsedBlood was tested for drug dependent platelet antibodies by Flow Cytometry at the Platelet and Neutrophil Immunology Laboratory at the Blood Center of Wisconsin.Summary of ResultsThe patient's serum showed evidence of drug dependent platelet antibodies to both oxaliplatin and irinotecan.ConclusionsDrug mediated immune thrombocytopenia is not uncommon. Time to severe acute thrombocytopenia and platelet recovery time varied post exposure of the drug. It is unclear whether steroid or IVIG administration had any effect on the platelet recovery time. Recovery from thrombocytopenia was observed in all 11 cases after the discontinuation of the insulting agent. Confirmation of the presence of drug dependent platelet antibodies against the chemotherapeutic agent by flow cytometry essential for diagnosis. This would be the first reported case of acute thrombocytopenia to two different chemotherapeutic agents in the same patient. Whether the reaction is two different mechanisms or if there is a cross reactivity between Oxaliplatin and Irinotecan has yet to be investigated.

Deviation from Guidelines in Use of Eltrombopag and Romiplostim in Clinical Practice for Children with Primary Immune Thrombocytopenia; Multi-Center Egyptian Experience

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 4907-4907
Author(s):  
Mohsen Saleh Elalfy ◽  
Tamer Hassan ◽  
Khalid Elsayh ◽  
Mohamed Maebid ◽  
Hoda Hassab ◽  
...  
Keyword(s):  
Adverse Events ◽  
Dose Escalation ◽  
Immune Thrombocytopenia ◽  
Conflicts Of Interest ◽  
Severe Bleeding ◽  
Inadequate Response ◽  
Serious Adverse Events ◽  
Platelet Counts ◽  
Chronic Itp ◽  
Primary Immune Thrombocytopenia

Background: Primary immune thrombocytopenia (ITP) has been treated with steroids or immune-suppressive. Thrombopoietin receptor agonists (TPO-RAs), changed the treatment landscape for ITP. Romiplostim and Eltrombopag were FDA approved for use in pediatric patients ≥1 year with ITP of >6 months' duration and insufficient response to previous therapy. Following the guidelines is not always feasible in practice. Aim and Methodology Centers treating more than 25 new cases/year with good recording system were included; aiming to report outcome, adverse events (AEs) and deviation from guidelines. Newly diagnosed ITP children were excluded. Data was collected from January 2017- June 2019; children age >1 to 18 years with persistent ITP (3-<12 months) or chronic (≥12 months), who had previously been treated with ≥1 previous ITP therapy, and had platelet counts (PC) persistently <30×109/l with moderate-severe bleeding. Eltrombopag was described in a dose of 25 mg/day for those > 1 to 12 years; or in 50 mg/day for those >12-18 years; dose was escalated to 50 or 75mg/day for those 6-12 and >12 years respectively on failure to have PC > 50 x 109/L. Weekly CBC and self-record of bleeding for 1st 24 week then bi-weekly. Failure to show a good response; Eltrombopag dosing should be adjusted to achieve that goal and not to exceed 200 × 109/L. Romiplostim was used in 1 ug/kg and escalated up to 10 ug/kg according to response. Serious AEs were reported to ethical committee within 24 hours. Results Two years Observational study of 204 out of 1080 primary ITP (268 persistent and 812 chronic); 55% females median age 8 years, received Eltrombopag (n=186), Romiplostim (n= 18) from 3-30 months was prescribed after failure of 1-5 lines of therapy in 15% of Persistent ITP and 20% of chronic ITP. Adequate response at 24 weeks in 60% on Eltrombopag or Romiplostim had a significant reduction in both skin-related and non-skin-related bleeding symptoms). However 50% discontinued treatment; (40% due to inadequate response, 8% irregular availability and 2% due to SAEs). Deviation from guidelines in 40%; (15% started Eltrombopag early between 3-6 months who showed a higher response rate; half of them could stop it without drop of PC); while 20% continued Eltrombopag for >12 month with lack of response after dose escalation at 24th week and 5% were not following the described dose or the regular monitoring. 40% of chronic ITP patients achieved platelet counts of >50 x 109/L or more at least twice. Common adverse events included; headache (10%). myalgia (10%), rhinitis (8%), diarrhea 8% Serious adverse events occurred in 4 (1 elevated transaminases, one unrelated life-threatening bleeding and two high PC; >1000 x109/L). Conclusion: TPO-RAs were used in almost 15% of persistent and 20% of chronic ITP as 2nd line or more therapy and in 90% was Eltrombopag; which produced an excellent response for those 3-6 months; deviating from guidelines. It showed a sustained platelet response in 40% of patients with chronic ITP, however it should be stopped if showing poor response after dose escalation. Disclosures No relevant conflicts of interest to declare.

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