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Shoko Noda-Narita, Keiichi Sumida, Akinari Sekine, Junichi Hoshino, Koki Mise, Tatsuya Suwabe, Noriko Hayami, Masayuki Yamanouchi, Toshiharu Ueno, Hiroki Mizuno, Masahiro Kawada, Rikako Hiramatsu, Eiko Hasegawa, Naoki Sawa, Kenmei Takaichi, Kenichi Ohashi, Takeshi Fujii, Yoshifumi Ubara
Thrombocytopenia, anasarca, fever, reticulin fibrosis, organomegaly (TAFRO) syndrome is a unique clinicopathologic subtype of multicentric Castleman's disease that has recently been identified in Japan. However, little is known about its renal histological changes and the optimal treatment for TAFRO syndrome. An 80-year-old Japanese woman was admitted to our hospital for evaluation of severe anasarca and weight gain (10 kg in a month). She had polyneuropathy, monoclonal plasma cell proliferative disorder with positive kappa M-protein, a sclerotic bone lesion, elevation of vascular endothelial growth factor (VEGF), skin changes, and extravascular volume overload, which fulfilled the diagnostic criteria for POEMS (polyneuropathy, organomegaly, endocrinopathy, and monoclonal protein, skin changes) syndrome...
February 21, 2018: CEN Case Reports
Jian-Chun Guo, Yi Zheng, Hai-Tao Chen, Haixia Zhou, Xian-Hui Huang, Li-Ping Zhong, Huai-Bin Zhou, Yu Huang, Dan-Li Xie, Yong-Liang Lou
Background and Aim: Thrombopoietin receptor agonists (TPO-RAs) have been shown to be safe and effective for adults with chronic immune thrombocytopenia (ITP). The aim of this meta-analysis is to assess the efficacy and safety of thrombopoietin receptor agonists for children with chronic ITP. Materials and Methods: Clinical randomized controlled trials (RCTs) evaluating the efficacy and safety of TPO-RAs in pediatric ITP patients published up to June 2017 were retrieved from PubMed, Cochrane Library, and Embase databases...
January 23, 2018: Oncotarget
Hagop M Kantarjian, Pierre Fenaux, Mikkael A Sekeres, Jeffrey Szer, Uwe Platzbecker, Andrea Kuendgen, Gianluca Gaidano, Wieslaw Wiktor-Jedrzejczak, Nancy Carpenter, Bhakti Mehta, Janet Franklin, Aristoteles Giagounidis
BACKGROUND: Treatment options for thrombocytopenia in myelodysplastic syndromes are scarce. As described previously in a randomised phase 2 study (n=250), 58 weeks of romiplostim treatment in patients with International Prognostic Scoring System (IPSS)-defined lower-risk (low-risk or intermediate-1 risk) myelodysplastic syndromes led to reduced platelet transfusions (p<0·0001) and increased International Working Group-defined haematological improvement-platelet rates (p<0·0001) versus placebo...
January 25, 2018: Lancet Haematology
Jeffrey A Gilreath, Mei Wei, Shilpa Paul, Charles J Parker, David D Stenehjem, George M Rodgers
Romiplostim is a thrombopoietin receptor agonist approved for the treatment of immune thrombocytopenia purpura. When following FDA-approved romiplostim prescribing recommendations to withhold treatment for platelet counts above 400k/µL, some patients exhibit a precipitous decline in their platelet count potentially causing patient harm. We present two cases where stable platelet counts were achieved only through persistent weekly dosing of romiplostim despite platelet counts above 400k/µL on the day of administration...
January 1, 2018: Journal of Oncology Pharmacy Practice
Jiaxing Zhang, Yi Liang, Yuan Ai, Xiaosi Li, Juan Xie, Youping Li, Wenyi Zheng, Rui He
In absence of direct comparison, we conducted an indirect-comparison meta-analysis to evaluate the efficacy and safety of thrombopoietin-receptor agonists(TPO-RAs) in treatment of pediatric persistent or chronic immune thrombocytopenia(ITP). PubMed, Embase, Cochrane Library, Clinical, China National Knowledge Infrastructure, and Chinese Biomedical Literature Database were searched from their earliest records to May 2017. Randomized controlled trials comparing the TPO-RAs with placebo in pediatric ITP were included...
January 12, 2018: Scientific Reports
Ryan K DasGupta, Lauren Levine, Tracy Wiczer, Spero Cataland
Background/rationale Romiplostim is a thrombopoietin receptor agonist recommended as a second-line therapy for immune thrombocytopenia. An initial dose of 1 mcg/kg/week subcutaneously with weekly 1 mcg/kg dose escalation is recommended per package labeling. Optimizing romiplostim dosing for hospitalized, corticosteroid- and intravenous immunoglobulin-refractory patients with severe thrombocytopenia secondary to immune thrombocytopenia may be critical for improving platelet responses, reducing the risk of bleeding, and decreasing hospital length of stay...
January 1, 2018: Journal of Oncology Pharmacy Practice
Hanny Al-Samkari, Ariela L Marshall, Katayoon Goodarzi, David J Kuter
No abstract text is available yet for this article.
December 14, 2017: Haematologica
Tianyi Li, Derek T Witteman, Eric D Weber, Warren L Alexander, John D Schaber
PURPOSE: To report a case of severe immune-mediated thrombocytopenia after intravitreal bevacizumab administration. METHODS: A 77-year-old man with right-sided macular degeneration received intravitreal bevacizumab. After his third treatment dose, he was hospitalized for symptomatic thrombocytopenia (platelet count of 3 k/μL) and underwent testing to determine the etiology. RESULTS: Initial platelet counts on admission were 3 k/μL, down from 238 k/μL 3 months before...
December 8, 2017: Retinal Cases & Brief Reports
Alessandro Pecci, Iman Ragab, Valeria Bozzi, Daniela De Rocco, Serena Barozzi, Tania Giangregorio, Heba Ali, Federica Melazzini, Mohamed Sallam, Caterina Alfano, Annalisa Pastore, Carlo L Balduini, Anna Savoia
Congenital amegakaryocytic thrombocytopenia (CAMT) is an inherited disorder characterized at birth by thrombocytopenia with reduced megakaryocytes, which evolves into generalized bone marrow aplasia during childhood. Although CAMT is genetically heterogeneous, mutations of MPL , the gene encoding for the receptor of thrombopoietin (THPO), are the only known disease-causing alterations. We identified a family with three children affected with CAMT caused by a homozygous mutation (p.R119C) of the THPO gene. Functional studies showed that p...
January 2018: EMBO Molecular Medicine
Xia Zhang, Yunhai Chuai, Wei Nie, Aiming Wang, Guanghai Dai
BACKGROUND: Chemotherapy-induced thrombocytopenia (CIT) is defined as a peripheral platelet count less than 100×109 /L, with or without bleeding in cancer patients receiving myelosuppressive chemotherapy. CIT is a significant medical problem during chemotherapy, and it carries the risk of sub-optimal overall survival and bleeding. Alternative interventions to platelet transfusion are limited. Different stages of preclinical and clinical studies have examined the thrombopoietin receptor agonists (TPO-RAs) for CIT in patients with solid tumours...
November 27, 2017: Cochrane Database of Systematic Reviews
Gürkan Bal, Depré Fabian, Dzamashvili Maia, Frauke Ringel, Abdulgabar Salama
The thrombopoietin receptor agonists (TPO-RAs), romiplostim and eltrombopag, stimulate megakaryopoiesis and thereby increase platelet counts. Both drugs are increasingly used in the treatment of immune thrombocytopenic purpura (ITP). To assess the effect of TPO-RAs on trilineage haematopoiesis, colony-forming cell (CFC) assays were performed on peripheral blood mononuclear cells of 8 healthy donors and 52 ITP patients. Additionally, we revaluated the regular and complete blood counts (CBCs) performed during romiplostim therapy in 45 patients and the CBCs performed in 9 patients during eltrombopag therapy...
December 2017: Annals of Hematology
Helga Dodillet, Karl-Anton Kreuzer, Ina Monsef, Nicole Skoetz
BACKGROUND: Myelodysplastic syndrome (MDS) is one of the most frequent haematologic malignancies of the elderly population and characterised by progenitor cell dysplasia with ineffective haematopoiesis and a high rate of transformation to acute myeloid leukaemia (AML). Thrombocytopenia represents a common problem for patients with MDS. ranging from mild to serious bleeding events and death. To manage thrombocytopenia, the current standard treatment includes platelet transfusion, unfortunately leading to a range of side effects...
September 30, 2017: Cochrane Database of Systematic Reviews
Prina Z Donga, Sara P Bilir, Gregg Little, Tim Babinchak, Julie Munakata
AIMS: Real-world evidence on the safety profile and costs associated with immune thrombocytopenic purpura (ITP) treatment in adults is lacking. This study quantifies and compares adverse event (AE) crude rates and costs associated with ITP treatments as found in claims data. MATERIALS AND METHODS: A retrospective claims-based analysis was conducted using IMS Pharmetrics Plus database. Included patients were ≥18 years old, with a diagnosis of ITP (2007-2012); an ITP-related claim for anti-D, intravenous immunoglobulin (IVIG), rituximab, romiplostim, or eltrombopag; and 1-year continuous enrollment (3-years for rituximab) during follow-up...
November 2017: Journal of Medical Economics
Jiaxing Zhang, Yi Liang, Yuan Ai, Juan Xie, Youping Li, Wenyi Zheng
OBJECTIVE: We conducted a systematic review to assess the efficacy and safety of Thrombopoietin-receptor agonists (TPOras) for pediatric immune thrombocytopenia (ITP). METHODS: We searched PubMed, Embase and Cochrane Library from their earliest records to January 2017. Randomized controlled trials (RCTs) were included. Primary outcomes were durable response and clinically significant bleeding. Secondary outcomes were overall response, overall bleeding events, the use of rescue medication and adverse events (AEs)...
October 2017: Expert Opinion on Pharmacotherapy
Sunil Lakhwani, María Perera, Fernando Fernández-Fuertes, Mario A Ríos de Paz, Melisa Torres, José María Raya, Miguel T Hernández
OBJECTIVE: To describe the reasons for and result of thrombopoietin receptor agonists (TPO-RA) switching in adult immune thrombocytopenia (ITP) patients of 4 Spanish centres. METHODS: We retrospectively analysed all patients who received sequential treatment with both TPO-RA between 2010 and 2015 recording clinical and biological parameters. RESULTS: Twenty-six patients were included; 17 received first romiplostim and 9 received first eltrombopag...
October 2017: European Journal of Haematology
Pierre Fenaux, Petra Muus, Hagop Kantarjian, Roger M Lyons, Richard A Larson, Mikkael A Sekeres, Pamela S Becker, Amelia Orejudos, Janet Franklin
Romiplostim can improve platelet counts in about 50% of patients with low- or intermediate 1-risk (lower risk) myelodysplastic syndromes (MDS) and thrombocytopenia, but its long-term toxicity and efficacy are not known. This open-label extension study evaluated the long-term safety and efficacy of romiplostim in 60 patients with lower risk MDS and platelet counts ≤50 × 109 /l. The primary endpoint was adverse event (AE) incidence. Secondary endpoints were efficacy parameters, including bleeding events and platelet response...
September 2017: British Journal of Haematology
Rena Matsumoto, Kazuhiro Ito, Naoko Hosono, Yasufumi Matsuda, Katsunori Tai, Ippei Sakamaki, Goh Aoki, Hirohito Yamazaki, Shinji Nakao, Takahiro Yamauchi
A 64-year-old man was diagnosed with acute myeloid leukemia M2 (FLT3-ITD-positive). After induction chemotherapy and four courses of consolidation therapy, he underwent umbilical cord blood transplantation (CBT) in his first remission. He developed acute graft-versus-host disease (skin stage 2) after successful engraftment. On post-transplantation day 147, he was admitted to the hospital suffering from pneumonia. During the treatment, drastic thrombocytopenia was observed on day 251. Both platelet-associated immunoglobulin G and platelet antibody producing B cells were detected, and he was diagnosed with immune thrombocytopenia (ITP)...
2017: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
Katherine A Sparger, Haley Ramsey, Viola Lorenz, Zhi-Jian Liu, Henry A Feldman, Nan Li, Tahirih Laforest, Martha C Sola-Visner
Thrombocytopenia is frequent among sick neonates. While most cases are transient, some neonates experience prolonged and severe thrombocytopenia. These infants often pose diagnostic and therapeutic challenges, and may receive large numbers of platelet transfusions. Romiplostim (ROM) is a thrombopoietin (TPO)-receptor-agonist approved for treatment of adults with chronic immune thrombocytopenia (ITP). The immature platelet fraction (IPF) is a novel measure of newly produced platelets, which could aid with the diagnostic evaluation of thrombocytopenic neonates...
May 26, 2017: Platelets
Douglas B Cines, Jeffrey Wasser, Francesco Rodeghiero, Beng H Chong, Michael Steurer, Drew Provan, Roger Lyons, Jaime Garcia-Chavez, Nancy Carpenter, Xuena Wang, Melissa Eisen
Primary immune thrombocytopenia is an autoimmune disorder characterized by increased platelet destruction and insufficient platelet production without another identified underlying disorder. Splenectomy may alter responsiveness to treatment and/or increase the risk of thrombosis, infection, and pulmonary hypertension. The analysis herein evaluated the safety and efficacy of the thrombopoietin receptor agonist romiplostim in splenectomized and nonsplenectomized adults with primary immune thrombocytopenia. Data were pooled across 13 completed clinical studies in adults with immune thrombocytopenia from 2002-2014...
August 2017: Haematologica
David Buchbinder, Diane Nugent, Loan Hsieh
Primary immune thrombocytopenia (ITP) is an autoimmune disorder characterized by isolated thrombocytopenia. In approximately one-third of cases, the duration of thrombocytopenia will extend beyond 12 months consistent with a diagnosis of chronic ITP. Minor bleeding manifestations are common in chronic ITP while severe or life-threatening bleeding complications are uncommon. Moreover, spontaneous resolution occurs in the majority of children with chronic ITP necessitating treatment in only those children with ongoing bleeding manifestations or impairment in health-related quality of life (HRQOL)...
2017: Drug Design, Development and Therapy
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