scholarly journals Safety and Efficacy of Thrombopoietin Analogs in the Treatment of Chronic Thrombocytopenic Purpura in Children: A Systematic Review

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4215-4215
Author(s):  
Usman Ali Akbar ◽  
Vivek Soorya Sathya Moorthy ◽  
Sudeep Yadav ◽  
Saman Bahram ◽  
Adnan Mehboob Qureshi ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Platelet Count ◽  
Placebo Group ◽  
Observational Study ◽  
Immune Thrombocytopenic Purpura ◽  
Research Funding ◽  
Pediatric Population ◽  
Platelet Response ◽  
Thrombocytopenic Purpura ◽  
Double Blinded

Abstract Introduction Immune thrombocytopenic purpura (ITP) is an autoimmune disorder consisting of low platelet count, purpura, and hemorrhagic episodes, caused by antiplatelet autoantibodies. Children who develop chronic or refractory ITP are at risk of bleeding after failing first-line therapies. Thrombopoietin analogs -Eltrombopag and romiplostim are safe and effective treatment options. Thrombopoietin receptor agonists (TPO-Ras) improve platelet production by activating the thrombopoietin pathway. They also stimulate megakaryocytes and hematopoietic stem cells. We analyzed the efficacy and toxicity of romiplostim and eltrombopag in chronic immune thrombocytopenic purpura in the pediatric population (1-17 years). Materials/Methods Following the PRISMA guidelines, we performed a comprehensive literature search on Pubmed, Embase, Cochrane Library, Web of Science, and Clinicaltrials.gov. We used the following keywords, "Thrombopoitein analogs", "TPOs", "Immune thrombocytopenic purpura", "Eltrombopag" and "Romiplostim" MeSh terms from the inception of data till 06/17/2021. We screened initial results from the search of 358 articles focusing on the pediatric population and finally included 9 clinical trials and 1 observational study. We excluded all case reports, case series, preclinical trials, review articles, and meta-analyses. We extracted the data for efficacy (platelet response, baseline platelet count, and Platelet count at first response) and safety (Bleeding or Grade ≥ 3 Adverse Events). Results: Romiplostim A total of 185 patients were analyzed in five clinical trials and 1 observational study employing Romiplostim in the treatment group. Platelet response (PR) (platelet count >50 × 109/L) has been reported in all the studies. In 3 randomized double-blinded control trials by Bussel 2011, Elfaly 2011, and Tarantino 2016, Romiplostim achieved a platelet response of 81.69% vs 12.9% in the placebo group. The other three studies reported substantial platelet response as stated in table 1. In all the studies 25 participants had a prior splenectomy. The most common side effects reported in the studies were bleeding (56.75%), headache (58.64%), contusion (50.76%), and epistaxis (49.23%). Clinically significant bleeding (grade 2-4) was reported by 2 studies in Romiplostim vs placebo group (71.08% vs 96%). Eltrombopag A total of 246 patients were analyzed in five clinical trials and 1 observational study. In a randomized double-blinded multicenter study by Grainger et al., Eltrombopag achieved a PR of 39.68% vs 3.4% in the placebo group. Other clinical trials reported a PR of 55.85% whereas the observational study by Neuner et al. reported a PR of 72% in the patient population. Clinically significant bleeding was reported by Grainger et al. and was 47 % in the eltrombopag group vs 7% in the placebo group. Fifteen patients in all the studies had a prior splenectomy. Conclusion: Thrombopoietin analogs such as romiplostim and eltrombopag show substantial platelet response and are associated with minimal side effects. However, more randomized clinical trials are needed to compare their head-to-head efficacy and safety in the treatment of chronic immune thrombocytopenic purpura in pediatric patients. Figure 1 Figure 1. Disclosures Anwer: Janssen pharmaceutical: Honoraria, Research Funding; GlaxoSmithKline: Research Funding; Allogene Therapeutics: Research Funding; BMS / Celgene: Honoraria, Research Funding.

Platelet Counts Following Eltrombopag Discontinuation in Patients with Chronic Immune Thrombocytopenic Purpura.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 3517-3517
Author(s):  
Gregory Cheng ◽  
Michael Tarantino ◽  
Terry Gernsheimer ◽  
Oliver Meyer ◽  
Andres Brainsky ◽  
...  
Keyword(s):  
Platelet Count ◽  
Immune Thrombocytopenic Purpura ◽  
Research Funding ◽  
Rescue Medication ◽  
Study Medication ◽  
Bleeding Events ◽  
Thrombocytopenic Purpura ◽  
Baseline Platelet Count ◽  
Platelet Counts ◽  
Transient Decrease

Abstract Abstract 3517 Poster Board III-454 BACKGROUND Eltrombopag (PROMACTA®; GlaxoSmithKline, Collegeville, PA, USA) is an oral, small molecule (565 Da), thrombopoietin receptor agonist that has been approved in the United States for the treatment of patients with chronic immune thrombocytopenic purpura (ITP). It is also being studied in thrombocytopenic patients with chronic liver disease, hepatitis C, myelodysplastic syndromes, and cancer. Withdrawal of treatments that stimulate platelet production may theoretically result in recurrent thrombocytopenia below pretreatment levels (below baseline). OBJECTIVE: To determine whether worsening of thrombocytopenia (ie, platelet count decrease below baseline) occurs after discontinuation of eltrombopag in patients with chronic ITP. METHODS: The lowest median platelet counts during the first 4 weeks posttherapy were compared with median baseline platelet counts. Data from 369 patients treated in 3 randomized, double-blind, placebo-controlled studies were analyzed: TRA100773A and TRA100773B were 6-week studies, and RAISE was a 6-month study. For all 3 studies, a baseline platelet count <30,000/μL was required. Platelet counts, bleeding events, and the use of ITP medication were examined in the 4 weeks following the discontinuation of eltrombopag or placebo. A transient decrease in platelet counts (ie, worsening of thrombocytopenia) was defined as a platelet count below 10,000/μL and at least 10,000/μL below each patient's baseline platelet count (Bussel N Eng J Med 2006). RESULTS: Using pooled data from the 3 studies, no decreases below baseline median platelet counts (placebo, 16,300/μL; eltrombopag, 16,000/μL) were observed compared to the lowest median platelet counts within the first 4 weeks posttherapy (placebo, 14,000/μL; eltrombopag, 17,000/μL). Across the pooled studies, a total of 10/128 (8%) of placebo-treated patients and 20/241 (8%) of eltrombopag-treated patients had a transient decrease in platelet counts in the 4 weeks following discontinuation or interruption of treatment. None of the 10 placebo-treated patients had bleeding events associated with posttreatment platelet nadirs. Three of the 20 eltrombopag-treated patients had bleeding events and/or rescue treatment associated with the platelet nadir in the 4-week posttreatment period. One patient discontinued eltrombopag after achieving platelet counts >200,000/μL following on-therapy rescue medication (corticosteroid 0.5 mg/kg/day); 9 days after discontinuing study medication, the patient had grade 1 gum bleeding and resumed daily corticosteroids at an increased dose. The second patient had grade 3 menorrhagia and was administered vincristine (patient had a history of similar symptoms). The third patient had Henoch-Schoenlein purpura, interrupted eltrombopag due to platelet counts >400,000/μL, and 7 days after holding eltrombopag had a platelet count of 2000/μL, experienced grade 1 mouth hemorrhage and grade 2 petechiae, and did not require rescue medication. The patient continued in the study for the full 6 months and following permanent discontinuation of eltrombopag, this patient did not experience a transient decrease in platelet counts or any bleeding. CONCLUSION: Across 3 placebo-controlled studies, the incidence of transient decreases in platelet counts following discontinuation or interruption of study medication was similar in patients receiving eltrombopag or placebo. Therefore, these decreases may be unrelated to study medication and may represent normal fluctuations in platelet counts in patients with chronic ITP. Transient platelet count decreases were generally not associated with bleeding events. Disclosures: Cheng: GlaxoSmithKline: Research Funding. Tarantino:GlaxoSmithKline: Speakers Bureau; Lundbeck: Speakers Bureau; Baxter: Membership on an entity's Board of Directors or advisory committees. Gernsheimer:GlaxoSmithKline: Honoraria, Research Funding; Amgen: Honoraria, Research Funding. Meyer:GlaxoSmithKline: Consultancy, Honoraria. Brainsky:GlaxoSmithKline: Employment. Stone:GlaxoSmithKline: Employment.

scholarly journals Immune Thrombocytopenic Purpura in Pregnancy- A Prospective Observational Study in a Tertiary Care Centre

2019 ◽  
Vol 34 (1) ◽  
pp. 15-21
Author(s):  
Tabassum Parveen ◽  
Firoza Begum ◽  
Nahreen Akhter ◽  
Nigar Sultana ◽  
Khairun Nahar
Keyword(s):  
Platelet Count ◽  
High Risk ◽  
Observational Study ◽  
Immune Thrombocytopenic Purpura ◽  
Platelet Transfusion ◽  
Prospective Observational Study ◽  
Thrombocytopenic Purpura ◽  
Neonatal Thrombocytopenia ◽  
Itp In Pregnancy ◽  
In Pregnancy

Objectives: Immune thrombocytopenic purpura (ITP) in pregnancy necessitates management of two patients, the mother and the newborn. Complications like maternal bleeding, fetal and neonatal thrombocytopenia demands appropriate and timely therapy. This prospective observational study was designed to explore and summarize the current approach to the investigation, diagnosis, management and outcome of ITP in pregnancy. Materials and Methods: Women with ITP admitted in the Fetomaternal Medicine Department of Bangabandhu Sheikh Mujib Medical University (BSMMU) from 2009 -2017, were included in the study. Total number of high risk pregnancy during that period were 7704 among them 20 cases were pregnancy with Immune Thrombocytopenic Purpura (ITP). Patients were managed under joint supervision of the fetomaternal medicine specialist and the hematologist. Prednisolone was considered as a first line drug in management protocol. Platelet transfusion was considered if there were symptoms or count <20X109/L at any stage of pregnancy or <50 X109 / L in late pregnancy without symptoms. Platelet count of newborn was performed at birth and repeated on day four and count<150X109/L was considered as neonatal thrombocytopenia. Results: Frequency of ITP among high risk patients was found 2.5/1000 live birth, most were preexisting (75%). Almost all cases (95%) were treated with prednisolone. Commonest clinical presentations were gum bleeding (70 %) and purpuric rashes (60%). Though during pregnancy, severe thrombocytopenia (<50 X109/L) was found in 7 patients (35%) but none was at the time of delivery, as drugs and/or platelet transfusion was considered to make delivery process safe. Platelet transfusion needed in 77.7% cases in a range of 1-75 units. Primary PPH noted in 3 cases (17%), increased bleeding during surgery in 5 patients (33%) and one patient needed ICU support. Neonatal thrombocytopenia noted in 5 cases (28%). Though 2 of the neonates needed NICU admission but none needed platelet transfusion and all the babies were discharged healthy. Conclusion: This study documents that pregnancy with ITP need close monitoring, require agents to raise the platelet count and repeated platelet transfusion to maintain reasonable safe platelet count. There are chances of PPH, capillary oozing during surgery. However good outcome is possible for most women, fetus and neonates with appropriate and timely therapy. Bangladesh J Obstet Gynaecol, 2019; Vol. 34(1): 15-21

Combination immunosuppressant therapy for patients with chronic refractory immune thrombocytopenic purpura

Blood ◽  
2010 ◽  
Vol 115 (1) ◽  
pp. 29-31 ◽  
Author(s):  
Donald M. Arnold ◽  
Ishac Nazi ◽  
Aurelio Santos ◽  
Howard Chan ◽  
Nancy M. Heddle ◽  
...  
Keyword(s):  
Platelet Count ◽  
Mycophenolate Mofetil ◽  
Adverse Events ◽  
Observational Study ◽  
Immune Thrombocytopenic Purpura ◽  
Treatment Options ◽  
Initial Response ◽  
Immunosuppressant Therapy ◽  
Thrombocytopenic Purpura ◽  
Refractory Itp

Abstract Treatment options for patients with chronic refractory immune thrombocytopenic purpura (ITP) are limited. Because combination immunosuppressant therapy appeared to be effective in ITP and other disorders, we used this approach in patients with particularly severe and refractory ITP. In this retrospective, observational study, we determined the response (platelet count above 30 × 109/L and doubling of baseline) among 19 refractory ITP patients. Treatment consisted of azathioprine, mycophenolate mofetil, and cyclosporine. The patients had failed a median of 6 prior treatments, including splenectomy (in all except 1). Of 19 patients, 14 (73.7%) achieved a response lasting a median of 24 months, after which time 8 (57.1%) relapsed. Of the 8 relapsing patients, 6 responded to additional treatments. Of the 14 patients who achieved an initial response, 2 (14.3%) remained in remission after eventually stopping all medications. Severe adverse events did not occur. Combination immunosuppressant therapy can produce a rise in the platelet count that is sometimes sustained in refractory ITP patients.

Helicobater Pylori Infection and Clinical Response to Eradication Therapy in Patients with Immune Thrombocytopenic Purpura (ITP).

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 3996-3996 ◽  
Author(s):  
Fabrizio Fabris ◽  
Emanuele Allemand ◽  
Raffaella Scandellari ◽  
Silvia Vettore ◽  
Maria Luigia Randi ◽  
...  
Keyword(s):  
Platelet Count ◽  
Immune Thrombocytopenic Purpura ◽  
Adult Population ◽  
Eradication Therapy ◽  
Geographic Area ◽  
Platelet Response ◽  
Thrombocytopenic Purpura ◽  
Stool Antigen Test ◽  
A Prospective Study ◽  
Negative Controls

Abstract We performed a prospective study in order to investigate the controversial association between HP infection and immune thrombocytopenic purpura (ITP). We studied the prevalence of HP infection and the efficacy of its eradication in 57 consecutive adult patients admitted to our ward for ITP. Thirty-three patients were females and 24 were males (median age 58 years; mean platelet count 68±42 x109/L). HP infection was demonstrated by the stool antigen test in 28 patients (49.1%). Such prevalence increased with the age. No statistical difference by gender, platelet count, disease duration, presence of specific platelet autoantibodies (MACE) and therapy regimens was observed between HP-positive and negative patients. On the other HP positive patients were significantly older than HP negative ones (62 versus 54 years, p<0,03) and presented more gastroenteric symptoms (32% versus 7%, p<0,05). Thirteen patients with chronic ITP were given HP eradication since their platelet count was above 20x109/L and they do not need for starting or modifying therapy of ITP for at least 6 months. HP eradication was performed by standard triple therapy based on omeprazole plus clarithromycin and amoxicillin for 1 week. The bacteria was eradicated in all cases. Twelve HP negative ITP patients were followed as controls. The mean platelet count was significantly increased in ITP patients after 3 and 6 months from HP eradication. However, considering as end-point, a platelet count increase of more than 50% over the basal value, the platelet response to the HP eradication appears to be better in eradicated patients only after 3 months of follow-up (83% of positive response versus 57%). We also studied in HP-positive patients the presence of antibodies against CagA citotoxic protein by ELISA method. Seventeen of the patients have anti-CagA antibodies (56%) but the positivity did not correlate with platelet response to the eradication. In conclusion the prevalence of HP infection in our patients with ITP was comparable to that observed in the adult population from the same geographic area. Infection is not associated with clinical or laboratory distinctive features with the exception of tan older age and gastroenteric symptoms. The eradication of the HP infection seems to improve mean platelet count in ITP patients but larger prospective studies are required to validate this approach. Platelet response to the HP eradication in HP positive ITP patients and in HP negative controls Platelets (x 109/L, mean ± SD) basal time 1 month 3 months 6 months 12 months * p<0.05 vs basal platelet count ITP Patients 13 HP+ eradicated 74±43 141±142 129±73* 104±54* 160±117 12 HP- controls (not eradicated) 62±35 76±33 73±40 78±51 76±48

Passive Transfer of Hepatitis B Core Antibody by Intravenous Immune Globulin in Patients with Immune Thrombocytopenic Purpura.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 2418-2418
Author(s):  
Donald M. Arnold ◽  
Julie Carruthers ◽  
Julie DiTomasso ◽  
Thea Gagliardi ◽  
Ralph M. Meyer ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Hepatitis B ◽  
Randomized Trial ◽  
Immune Globulin ◽  
Immune Thrombocytopenic Purpura ◽  
Research Funding ◽  
Passive Transfer ◽  
High Rate ◽  
Intravenous Immune Globulin ◽  
Thrombocytopenic Purpura

Abstract Abstract 2418 Poster Board II-395 Background: Rituximab may reactivate remote hepatitis B infection. Thus, hepatitis B core antibody (HBcAb) seropositivity was an exclusion for a randomized trial of rituximab in patients with immune thrombocytopenic purpura (ITP) in Canada. During screening for this trial, we observed a high rate of HBcAb seropositivity at our centre. We investigated passive antibody transfer by intravenous immune globulin (IVIg) as a potential explanation. Methods: We performed a case-control study of consecutive ITP patients screened for eligibility for a randomized trial of rituximab. We compared the frequency of prior IVIg use and the IVIg product administered to patients who tested positive (n=11) and negative (n=13) for HBcAb. Total (IgG and IgM) HBcAb was tested using a microparticle enzyme immunoassay (MEIA) that measures the rate of inhibition compared with an internal cut-off value (AxSYM®, Abbott Laboratories, IL, USA). We calculated the odds ratio (OR) and 95% confidence interval (CI) for IVIg exposure. We also tested different lots of 2 IVIg products directly for HBcAb in the MEIA. Results: Of 24 consecutive patients with ITP screened, 11 (45.8%) were positive for HBcAb and thus were excluded from the randomized trial. The observed rate of HBcAb seropositivity in this cohort was 35 times higher than the expected seroprevalence in Canada of 1.3% (O'Brien Transfusion 2009). Of 11 HBcAb-positive patients, 10 (90.9%) had received prior IVIg [OR, 16 (95% CI, 1.54, 166.05)]. Of the 10 patients who received IVIg, 8 (80.0%) received Gamunex (10% human immune globulin, Talecris Biotherapeutics, Clayton, NC). HBcAb was repeated in 8 seropositive patients, of whom 4 were negative on repeat testing 7 to 104 weeks after the last dose of IVIg. Of the 13 HBcAb-negative patients, 5 (38.5%) had received prior IVIg; none were Gamunex. Seroconversion was documented in one patient serially tested after receiving Gamunex; MEIA rates decreased (became positive) rapidly, then gradually increased to normal after 7 weeks. Samples from 4 lots of Gamunex were reactive in the MEIA; whereas 3 lots of IGIVnex (Talecris Biotherapeutics) were non-reactive. Conclusions: False positive HBcAb results may occur due to the passive transfer of HBcAb from Gamunex, an IVIg product manufactured from donors who are not screened for HBcAb. HBcAb should be interpreted cautiously in any patient who received IVIg. We recommend HBcAb testing before IVIg administration so that treatments such as rituximab are not withheld unnecessarily. Disclosures: Arnold: Hoffman-LaRoche: Research Funding; Amgen: Consultancy, Honoraria. Meyer:The NCIC CTG is a clinical trials cooperative group. It receives its base grant support from the Canadian Cancer Society Research Institute. Many of its clinical trials include research support from industry. : Research Funding.

Clinical Features, Treatment, and Outcome of Severe HIV-Associated Immune Thrombocytopenic Purpura In the HAART Era

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 2522-2522
Author(s):  
Kimberley LS Ambler ◽  
Linda M Vickars ◽  
Chantal S Leger ◽  
Lynda M Foltz ◽  
Julio SG Montaner ◽  
...  
Keyword(s):  
Platelet Count ◽  
Hepatitis C ◽  
Hepatitis B ◽  
Clinical Features ◽  
Hiv Risk ◽  
Immune Thrombocytopenic Purpura ◽  
Red Blood Cell Transfusion ◽  
Platelet Response ◽  
Thrombocytopenic Purpura ◽  
History Of

Abstract Abstract 2522 Background: The association between HIV and immune thrombocytopenic purpura (ITP) is well documented. Although HIV-associated ITP responds both to highly active anti-retroviral therapy (HAART) and treatments used in classic ITP, the clinical features of HIV-associated ITP were documented prior to the widespread use of HAART, and there are currently no widely accepted guidelines for the management of HIV-associated ITP. Here we describe the clinical features, treatment, and outcomes of patients diagnosed with severe HIV-associated ITP in the HAART era. Methods: We searched the BC Centre for Excellence in HIV/AIDS (CFE) database to identify patients with ≥ 1 platelet count <20 × 109/L since January 1996, the year HAART was widely adopted in British Columbia. The cutoff value of <20 × 109/L was chosen as a clinically relevant platelet count since these patients generally require treatment. Patient charts were reviewed, clinical data extracted, and only patients with a diagnosis of ITP made by a hematologist were included in the analysis. Descriptive statistics were used to summarize the data. Results: Of 8922 patients in the CFE database since 1996, 31 (0.3%) with a diagnosis of ITP and a platelet count <20 × 109/L were identified. The median age at ITP diagnosis was 37 (range 27–66) years and 25 (81%) were male. The median platelet count was 6 (2-19) × 109/L and median hemoglobin 129 (34-165) g/L. Eighteen patients (51%) presented with clinical bleeding and 4 (13%) required packed red blood cell transfusion. Sixteen (62%) of 26 patients with a documented HIV risk factor had a history of injection drug use (IDU). Four patients (13%) were coinfected with the hepatitis B virus and 12 (39%) with hepatitis C. At ITP diagnosis, 8 of 29 patients (28%) had a CD4 count <200 cells/mL and the median CD4 was 290 (20-600) cells/mL; 5 had a prior AIDS-defining opportunistic infection or neoplasm. Although 29 patients received antiretrovirals at some point, only 10 (32%) were receiving HAART at ITP diagnosis. A bone marrow aspirate and biopsy was performed in 6 patients (19%) and was consistent with ITP in all. Initial ITP treatment included: IVIG, n=12; steroids, n=10; anti-RhD, n=8; HAART, n=3. The median number of treatments received was 1 (0-3) and median time to a platelet count >20 × 109/L was 13.5 (1-1379) days. Median platelet response within 30 days was 58 (5-322) × 109/L (n=26) but only 3 patients (10%) achieved a platelet count in the normal range. At a median follow-up of 48 (0.2-138) months, 27 patients (87%) required secondary ITP treatment for a recurrent platelet count <20 × 109/L; median 10 (5-20) × 109/L, including 8 of 13 patients receiving HAART with initial ITP therapy. Secondary ITP treatment included: IVIG, n=9; anti-RhD, n=6; steroids, n=4; splenectomy, n=3; danazol, n=1; and HAART, n=1. Median platelet response to secondary treatment was 42 (21-198) × 109/L. Response to ITP treatment was not significantly associated with treatment received but was lower in the following patients: 4/15 with comorbidities (5 related to the liver) vs 10/16 without; 1/21 IDU vs 4/10 with sexual HIV risk; and 4/13 with hepatitis B or C coinfection vs 13/16 without (p<0.05 for all). Complications of ITP treatment occurred in 2 patients: psychiatric effect of steroids, n=1; and post-splenectomy fever and hematoma, n=1. There were 4 deaths, causes were: GI bleed, n=2; Evan's syndrome and hepatic failure, n=1; advanced HIV, n=1. Of the 4 patients that died, 3 had a history of IDU. Comorbidities in patients who died included: hepatitis C, n=3; hepatic cirrhosis, n=2; portal hypertension, n=1; hepatocellular failure, n=1. One patient who died of GI bleeding had a history of IDU, hepatitis C coinfection, and died of variceal bleeding despite a normal platelet count following splenectomy. Conclusions: Most patients with severe HIV-associated ITP diagnosed in the HAART era achieved a safe platelet count with primary ITP treatment and there were few treatment complications. However, nearly all required retreatment for severe ITP, including 8 of 13 patients receiving HAART with initial ITP therapy. Inferior platelet response was associated with a history of IDU, comorbidities, and hepatitis B or C coinfection, and 3 of 4 deaths occurred in patients with a history of IDU, therefore new approaches to the treatment of severe ITP in this patient population are needed. This is to our knowledge the largest series of HIV-associated ITP reported in the era of HAART. Disclosures: No relevant conflicts of interest to declare.

Efficacy and Safety of a New Intravenous Immunoglobulin Product in Patients with Chronic Immune Thrombocytopenic Purpura.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 1307-1307
Author(s):  
Tadeusz Robak ◽  
Abdulgabar Salama ◽  
Lidia Kovaleva ◽  
Yaroslava I. Vyhovska ◽  
Simon Davies ◽  
...  
Keyword(s):  
Platelet Count ◽  
Adverse Events ◽  
Intravenous Immunoglobulin ◽  
Primary Endpoint ◽  
Immune Thrombocytopenic Purpura ◽  
Efficacy And Safety ◽  
Platelet Response ◽  
Thrombocytopenic Purpura ◽  
Platelet Counts ◽  
Chronic Itp

Abstract Background Intravenous immunoglobulin (IVIG) is an accepted treatment for immune thrombocytopenic purpura (ITP). A new liquid 10% human IgG preparation stabilized with proline at a pH of 4.8 (trade name: Privigen) was recently developed. It can be stored at room temperature and is therefore always ready to use. Here we report its efficacy and safety in patients with chronic ITP. Patients and Methods Fifty-seven patients with chronic ITP and a platelet count below 20 x 109/L were included in this open-label, single arm, multi-center Phase III trial. IVIG was given at a dose of 1 g/kg on 2 consecutive days at a maximum infusion rate of 4 mg/kg/min. A subset (56.1%) of the patients received premedication (acetaminophen or diphenhydramine) to avoid adverse events. The primary endpoint was the platelet response rate, defined as the percentage of patients showing an increase in platelet count to ≥50 x 109/L within 7 days of the first infusion. Secondary endpoints included platelet counts at specified time points, time to platelet response, duration of platelet response, and regression of hemorrhages at different bleeding sites. Safety was evaluated by the frequency and severity of adverse events. Results The primary endpoint, an increase in platelet counts to ≥50 x 109/L, was achieved by 81% of the subjects (95% CI: 69–89%). The highest median platelet count (149 x 109/L) was observed on day 8. Median time to response was 2.5 days, with 43% of subjects responding within 1 day. Median duration of platelet response (days with platelet count ≥ 50 x 109/L) was 15.4 days. Regression rates for various bleeding sites ranged from 78% to 100%. Regression of bleeding correlated with increases in platelet counts. Adverse events were reported in 52 (91%) subjects. The most common adverse event was headache (67%), the incidence and severity of which was attenuated by premedication with acetaminophen/diphenhydramine. There were 3 serious adverse events, one of which (aseptic meningitis) was considered related to the study medication. Conclusions The present study has shown the safety and significant efficacy of a novel, 10% liquid, ready for use IVIG preparation, in patients with chronic ITP. A rapid increase in platelet counts to a level where severe bleeding episodes become more unlikely was seen in the majority of patients, as expected with IVIG given at 1 g/kg on 2 consecutive days. The increase in platelet counts was associated with a regression of bleeding. Adverse events were generally mild to moderate in severity, corresponded to those expected with IVIG, and could be prevented with premedication.

Treatment Does Not Correlate with Quality of Life in Children with Immune Thrombocytopenic Purpura: Results From the KIT International Cross-Cultural Validation Study.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 2501-2501
Author(s):  
Robert Klaassen ◽  
John D. Grainger ◽  
Arne Riedlinger ◽  
Victor S. Blanchette ◽  
Tricia Burke ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Platelet Count ◽  
Multiple Regression ◽  
Immune Thrombocytopenic Purpura ◽  
Research Funding ◽  
Country Of Origin ◽  
Self Report ◽  
Chronic Patients ◽  
Thrombocytopenic Purpura

Abstract Abstract 2501 Poster Board II-478 Introduction: There is considerable controversy surrounding whether or not children with Immune Thrombocytopenic Purpura (ITP) should be treated if they present without bleeding. One of the potential benefits of treatment would be to improve the child's health-related quality of life (HRQoL). Patients and Methods: Variables including age, sex, type of ITP(acute versus chronic), treatment (observation, IVIG, Anti-D, and prednisone), platelet count and country of origin were analysed by multiple regression to determine their relationship to HRQoL as measured by the Kid's ITP Tools (KIT) child self-report version. Results: 77 children from Uruguay (n=15), France (n=25), Germany (n=13) and the UK (n=24) self-completed the KIT. Mean platelet counts were: 6 for acute ITP patients (mean age 8.6 yrs) and 30 for chronic patients (mean age of 10.8 years). KIT scores by type of ITP and country are shown in the Figure. Multiple regression found that only the type of ITP (p=0.04) and the country of origin (p=0.029) were significantly associated with the KIT scores. Age, sex, platelet count and treatment were not correlated with KIT scores (p>0.16). Conclusion: The method of treatment did not have a significant impact on child-reported HRQoL. However, differences in HRQoL scores were found between acute and chronic groups and by country of origin. Disclosures: Klaassen: Cangene : Research Funding. Blanchette:Cangene: Research Funding. Young:Cangene: Research Funding.

Helicobacter pylori infection and chronic immune thrombocytopenic purpura: long-term results of bacterium eradication and association with bacterium virulence profiles

Blood ◽  
2007 ◽  
Vol 110 (12) ◽  
pp. 3833-3841 ◽  
Author(s):  
Giovanni Emilia ◽  
Mario Luppi ◽  
Patrizia Zucchini ◽  
Monica Morselli ◽  
Leonardo Potenza ◽  
...  
Keyword(s):  
Helicobacter Pylori ◽  
Chronic Gastritis ◽  
Immune Thrombocytopenic Purpura ◽  
Long Term Results ◽  
Platelet Response ◽  
Related Factors ◽  
Thrombocytopenic Purpura ◽  
Platelet Recovery ◽  
Chronic Immune Thrombocytopenic Purpura ◽  
H Pylori

AbstractEradication of Helicobacter pylori may lead to improvement of chronic immune thrombocytopenic purpura (ITP), although its efficacy over time is uncertain. We report the results of H pylori screening and eradication in 75 consecutive adult patients with ITP. We also used molecular methods to investigate lymphocyte clonality and H pylori genotypes in the gastric biopsies from 10 H pylori–positive patients with ITP and 19 H pylori–positive patients without ITP with chronic gastritis. Active H pylori infection was documented in 38 (51%) patients and successfully eradicated in 34 (89%) patients. After a median follow-up of 60 months, a persistent platelet response in 23 (68%) of patients with eradicated infection was observed; 1 relapse occurred. No differences in mucosal B- or T-cell clonalities were observed between patients with ITP and control participants. Of note, the frequency of the H pylori cagA gene (P = .02) and the frequency of concomitant H pylori cagA, vacAs1, and iceA genes (triple-positive strains; P = .015) resulted statistically higher in patients with ITP than in control participants. All asymptomatic H pylori–positive patients with ITP were suffering from chronic gastritis. Our data suggest a sustained platelet recovery in a proportion of patients with ITP by H pylori eradication alone. Overrepresentation of specific H pylori genotypes in ITP suggests a possible role for bacterium-related factors in the disease pathogenesis.

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