Long-term safety and effectiveness of romiplostim for chronic idiopathic thrombocytopenic purpura in real-world settings
Idiopathic thrombocytopenic purpura (ITP), an autoimmune hematologic disorder characterized by severe platelet count reduction, can be treated with romiplostim. However, post-marketing safety and effectiveness data for romiplostim in Japan are scarce. This prospective, observational, post-marketing...
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| Veröffentlicht in: | International journal of hematology 2024-12, Vol.120 (6), p.665-674 |
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| creator | Obara, Naoshi Hatanaka, Shigeki Tsuji, Yukie Higashi, Koji |
| description | Idiopathic thrombocytopenic purpura (ITP), an autoimmune hematologic disorder characterized by severe platelet count reduction, can be treated with romiplostim. However, post-marketing safety and effectiveness data for romiplostim in Japan are scarce. This prospective, observational, post-marketing Specified Use-Results Survey evaluated the real-world safety and effectiveness of romiplostim for 2 years. All patients treated with romiplostim during the survey period were eligible. Of the 1622 patients in the safety analysis set, 94.08% (1526/1622) had chronic ITP. The mean single dose of romiplostim was stable after 12 weeks and remained |
| doi_str_mv | 10.1007/s12185-024-03847-4 |
| format | Article |
| fullrecord | <record><control><sourceid>proquest_pubme</sourceid><recordid>TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_11588964</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>3110914838</sourcerecordid><originalsourceid>FETCH-LOGICAL-c400t-e2ab3a4174516448f32285434e3c2b0a2507a38b8fc14afa136e0326e79d7d5b3</originalsourceid><addsrcrecordid>eNp9kUuLFDEUhQtRnHb0D7iQgBs30Twr6ZXI4Asa3Og6pFI33RmqkjJJjfa_N22P42MhBG7I-e7JvZyue0rJS0qIelUoo1piwgQmXAuFxb1uQ3UvMVdK3O82ZMskloqSi-5RKdeEUEWEethd8C0XTAqx6b7vUtzjCnlGxXqoR2TjiMB7cDXcQIRSUPIopzksUyo1zMinjNwhpxgcCmNIi62Hdq3taR6SO9a0wElb1tyORSGiDHbC31KeRlSg1hD35XH3wNupwJPbetl9eff289UHvPv0_uPVmx12gpCKgdmBW0GVkLQXQnvOmJaCC-CODcQySZTletDeUWG9pbwHwlkPajuqUQ78snt99l3WYYbRQazZTmbJYbb5aJIN5m8lhoPZpxtDqdR624vm8OLWIaevK5Rq5lAcTJONkNZiOKVkS4XmuqHP_0Gv05pj269RnClGFGWNYmfK5VRKBn83DSXmlKw5J2tasuZnsuY0xbM_97hr-RVlA_gZKE2Ke8i___6P7Q9jjbFr</addsrcrecordid><sourcetype>Open Access Repository</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>3132720712</pqid></control><display><type>article</type><title>Long-term safety and effectiveness of romiplostim for chronic idiopathic thrombocytopenic purpura in real-world settings</title><source>MEDLINE</source><source>SpringerNature Journals</source><creator>Obara, Naoshi ; Hatanaka, Shigeki ; Tsuji, Yukie ; Higashi, Koji</creator><creatorcontrib>Obara, Naoshi ; Hatanaka, Shigeki ; Tsuji, Yukie ; Higashi, Koji</creatorcontrib><description>Idiopathic thrombocytopenic purpura (ITP), an autoimmune hematologic disorder characterized by severe platelet count reduction, can be treated with romiplostim. However, post-marketing safety and effectiveness data for romiplostim in Japan are scarce. This prospective, observational, post-marketing Specified Use-Results Survey evaluated the real-world safety and effectiveness of romiplostim for 2 years. All patients treated with romiplostim during the survey period were eligible. Of the 1622 patients in the safety analysis set, 94.08% (1526/1622) had chronic ITP. The mean single dose of romiplostim was stable after 12 weeks and remained < 6 μg/kg in approximately 70% of patients until 104 weeks. Within 2 years, 14.92% of patients discontinued romiplostim because of adverse events, while 6.47% discontinued because of suspected adverse drug reactions. In contrast, 14.00% of patients discontinued romiplostim because of symptom improvement. Before romiplostim initiation, platelet count was < 2.0 × 10
4
/µL in 60.54% of patients, and the mean platelet count was 2.84 ± 5.76 × 10
4
/µL. Platelet count was 9.19 ± 13.01 × 10
4
/µL after 4 weeks, and remained between 10.34 ± 10.72 and 12.38 ± 12.63 × 10
4
/µL from 8 to 104 weeks of treatment. No specific concerns were revealed regarding the safety and effectiveness of romiplostim in chronic ITP; the findings demonstrated a favorable risk–benefit balance for romiplostim in this population.
Trial registration
: UMIN000047864 (
www.umin.ac.jp/ctr
).</description><identifier>ISSN: 0925-5710</identifier><identifier>ISSN: 1865-3774</identifier><identifier>EISSN: 1865-3774</identifier><identifier>DOI: 10.1007/s12185-024-03847-4</identifier><identifier>PMID: 39342544</identifier><language>eng</language><publisher>Singapore: Springer Nature Singapore</publisher><subject>Adolescent ; Adult ; Aged ; Aged, 80 and over ; Chronic Disease ; Effectiveness ; Female ; Hematological diseases ; Hematology ; Humans ; Idiopathic thrombocytopenic purpura ; Japan ; Male ; Marketing ; Medicine ; Medicine & Public Health ; Middle Aged ; Oncology ; Original ; Original Article ; Platelet Count ; Platelets ; Product Surveillance, Postmarketing ; Prospective Studies ; Purpura ; Purpura, Thrombocytopenic, Idiopathic - drug therapy ; Receptors, Fc - administration & dosage ; Receptors, Fc - therapeutic use ; Recombinant Fusion Proteins - administration & dosage ; Recombinant Fusion Proteins - adverse effects ; Recombinant Fusion Proteins - therapeutic use ; Safety ; Side effects ; Surveys ; Thrombocytopenic purpura ; Thrombopoietin - administration & dosage ; Thrombopoietin - adverse effects ; Thrombopoietin - therapeutic use ; Time Factors ; Treatment Outcome ; UMIN ; UMIN000047864 ; Young Adult</subject><ispartof>International journal of hematology, 2024-12, Vol.120 (6), p.665-674</ispartof><rights>The Author(s) 2024</rights><rights>2024. The Author(s).</rights><rights>The Author(s) 2024. This work is published under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.</rights><rights>The Author(s) 2024 2024</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><cites>FETCH-LOGICAL-c400t-e2ab3a4174516448f32285434e3c2b0a2507a38b8fc14afa136e0326e79d7d5b3</cites><orcidid>0000-0001-6563-3093</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://link.springer.com/content/pdf/10.1007/s12185-024-03847-4$$EPDF$$P50$$Gspringer$$Hfree_for_read</linktopdf><linktohtml>$$Uhttps://link.springer.com/10.1007/s12185-024-03847-4$$EHTML$$P50$$Gspringer$$Hfree_for_read</linktohtml><link.rule.ids>230,314,780,784,885,27924,27925,41488,42557,51319</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/39342544$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Obara, Naoshi</creatorcontrib><creatorcontrib>Hatanaka, Shigeki</creatorcontrib><creatorcontrib>Tsuji, Yukie</creatorcontrib><creatorcontrib>Higashi, Koji</creatorcontrib><title>Long-term safety and effectiveness of romiplostim for chronic idiopathic thrombocytopenic purpura in real-world settings</title><title>International journal of hematology</title><addtitle>Int J Hematol</addtitle><addtitle>Int J Hematol</addtitle><description>Idiopathic thrombocytopenic purpura (ITP), an autoimmune hematologic disorder characterized by severe platelet count reduction, can be treated with romiplostim. However, post-marketing safety and effectiveness data for romiplostim in Japan are scarce. This prospective, observational, post-marketing Specified Use-Results Survey evaluated the real-world safety and effectiveness of romiplostim for 2 years. All patients treated with romiplostim during the survey period were eligible. Of the 1622 patients in the safety analysis set, 94.08% (1526/1622) had chronic ITP. The mean single dose of romiplostim was stable after 12 weeks and remained < 6 μg/kg in approximately 70% of patients until 104 weeks. Within 2 years, 14.92% of patients discontinued romiplostim because of adverse events, while 6.47% discontinued because of suspected adverse drug reactions. In contrast, 14.00% of patients discontinued romiplostim because of symptom improvement. Before romiplostim initiation, platelet count was < 2.0 × 10
4
/µL in 60.54% of patients, and the mean platelet count was 2.84 ± 5.76 × 10
4
/µL. Platelet count was 9.19 ± 13.01 × 10
4
/µL after 4 weeks, and remained between 10.34 ± 10.72 and 12.38 ± 12.63 × 10
4
/µL from 8 to 104 weeks of treatment. No specific concerns were revealed regarding the safety and effectiveness of romiplostim in chronic ITP; the findings demonstrated a favorable risk–benefit balance for romiplostim in this population.
Trial registration
: UMIN000047864 (
www.umin.ac.jp/ctr
).</description><subject>Adolescent</subject><subject>Adult</subject><subject>Aged</subject><subject>Aged, 80 and over</subject><subject>Chronic Disease</subject><subject>Effectiveness</subject><subject>Female</subject><subject>Hematological diseases</subject><subject>Hematology</subject><subject>Humans</subject><subject>Idiopathic thrombocytopenic purpura</subject><subject>Japan</subject><subject>Male</subject><subject>Marketing</subject><subject>Medicine</subject><subject>Medicine & Public Health</subject><subject>Middle Aged</subject><subject>Oncology</subject><subject>Original</subject><subject>Original Article</subject><subject>Platelet Count</subject><subject>Platelets</subject><subject>Product Surveillance, Postmarketing</subject><subject>Prospective Studies</subject><subject>Purpura</subject><subject>Purpura, Thrombocytopenic, Idiopathic - drug therapy</subject><subject>Receptors, Fc - administration & dosage</subject><subject>Receptors, Fc - therapeutic use</subject><subject>Recombinant Fusion Proteins - administration & dosage</subject><subject>Recombinant Fusion Proteins - adverse effects</subject><subject>Recombinant Fusion Proteins - therapeutic use</subject><subject>Safety</subject><subject>Side effects</subject><subject>Surveys</subject><subject>Thrombocytopenic purpura</subject><subject>Thrombopoietin - administration & dosage</subject><subject>Thrombopoietin - adverse effects</subject><subject>Thrombopoietin - therapeutic use</subject><subject>Time Factors</subject><subject>Treatment Outcome</subject><subject>UMIN</subject><subject>UMIN000047864</subject><subject>Young Adult</subject><issn>0925-5710</issn><issn>1865-3774</issn><issn>1865-3774</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2024</creationdate><recordtype>article</recordtype><sourceid>C6C</sourceid><sourceid>EIF</sourceid><recordid>eNp9kUuLFDEUhQtRnHb0D7iQgBs30Twr6ZXI4Asa3Og6pFI33RmqkjJJjfa_N22P42MhBG7I-e7JvZyue0rJS0qIelUoo1piwgQmXAuFxb1uQ3UvMVdK3O82ZMskloqSi-5RKdeEUEWEethd8C0XTAqx6b7vUtzjCnlGxXqoR2TjiMB7cDXcQIRSUPIopzksUyo1zMinjNwhpxgcCmNIi62Hdq3taR6SO9a0wElb1tyORSGiDHbC31KeRlSg1hD35XH3wNupwJPbetl9eff289UHvPv0_uPVmx12gpCKgdmBW0GVkLQXQnvOmJaCC-CODcQySZTletDeUWG9pbwHwlkPajuqUQ78snt99l3WYYbRQazZTmbJYbb5aJIN5m8lhoPZpxtDqdR624vm8OLWIaevK5Rq5lAcTJONkNZiOKVkS4XmuqHP_0Gv05pj269RnClGFGWNYmfK5VRKBn83DSXmlKw5J2tasuZnsuY0xbM_97hr-RVlA_gZKE2Ke8i___6P7Q9jjbFr</recordid><startdate>202412</startdate><enddate>202412</enddate><creator>Obara, Naoshi</creator><creator>Hatanaka, Shigeki</creator><creator>Tsuji, Yukie</creator><creator>Higashi, Koji</creator><general>Springer Nature Singapore</general><general>Springer Nature B.V</general><scope>C6C</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7T5</scope><scope>7T7</scope><scope>7TM</scope><scope>8FD</scope><scope>C1K</scope><scope>FR3</scope><scope>H94</scope><scope>K9.</scope><scope>NAPCQ</scope><scope>P64</scope><scope>7X8</scope><scope>5PM</scope><orcidid>https://orcid.org/0000-0001-6563-3093</orcidid></search><sort><creationdate>202412</creationdate><title>Long-term safety and effectiveness of romiplostim for chronic idiopathic thrombocytopenic purpura in real-world settings</title><author>Obara, Naoshi ; Hatanaka, Shigeki ; Tsuji, Yukie ; Higashi, Koji</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c400t-e2ab3a4174516448f32285434e3c2b0a2507a38b8fc14afa136e0326e79d7d5b3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2024</creationdate><topic>Adolescent</topic><topic>Adult</topic><topic>Aged</topic><topic>Aged, 80 and over</topic><topic>Chronic Disease</topic><topic>Effectiveness</topic><topic>Female</topic><topic>Hematological diseases</topic><topic>Hematology</topic><topic>Humans</topic><topic>Idiopathic thrombocytopenic purpura</topic><topic>Japan</topic><topic>Male</topic><topic>Marketing</topic><topic>Medicine</topic><topic>Medicine & Public Health</topic><topic>Middle Aged</topic><topic>Oncology</topic><topic>Original</topic><topic>Original Article</topic><topic>Platelet Count</topic><topic>Platelets</topic><topic>Product Surveillance, Postmarketing</topic><topic>Prospective Studies</topic><topic>Purpura</topic><topic>Purpura, Thrombocytopenic, Idiopathic - drug therapy</topic><topic>Receptors, Fc - administration & dosage</topic><topic>Receptors, Fc - therapeutic use</topic><topic>Recombinant Fusion Proteins - administration & dosage</topic><topic>Recombinant Fusion Proteins - adverse effects</topic><topic>Recombinant Fusion Proteins - therapeutic use</topic><topic>Safety</topic><topic>Side effects</topic><topic>Surveys</topic><topic>Thrombocytopenic purpura</topic><topic>Thrombopoietin - administration & dosage</topic><topic>Thrombopoietin - adverse effects</topic><topic>Thrombopoietin - therapeutic use</topic><topic>Time Factors</topic><topic>Treatment Outcome</topic><topic>UMIN</topic><topic>UMIN000047864</topic><topic>Young Adult</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Obara, Naoshi</creatorcontrib><creatorcontrib>Hatanaka, Shigeki</creatorcontrib><creatorcontrib>Tsuji, Yukie</creatorcontrib><creatorcontrib>Higashi, Koji</creatorcontrib><collection>Springer Nature OA Free Journals</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>Immunology Abstracts</collection><collection>Industrial and Applied Microbiology Abstracts (Microbiology A)</collection><collection>Nucleic Acids Abstracts</collection><collection>Technology Research Database</collection><collection>Environmental Sciences and Pollution Management</collection><collection>Engineering Research Database</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>Nursing & Allied Health Premium</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>MEDLINE - Academic</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>International journal of hematology</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Obara, Naoshi</au><au>Hatanaka, Shigeki</au><au>Tsuji, Yukie</au><au>Higashi, Koji</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Long-term safety and effectiveness of romiplostim for chronic idiopathic thrombocytopenic purpura in real-world settings</atitle><jtitle>International journal of hematology</jtitle><stitle>Int J Hematol</stitle><addtitle>Int J Hematol</addtitle><date>2024-12</date><risdate>2024</risdate><volume>120</volume><issue>6</issue><spage>665</spage><epage>674</epage><pages>665-674</pages><issn>0925-5710</issn><issn>1865-3774</issn><eissn>1865-3774</eissn><abstract>Idiopathic thrombocytopenic purpura (ITP), an autoimmune hematologic disorder characterized by severe platelet count reduction, can be treated with romiplostim. However, post-marketing safety and effectiveness data for romiplostim in Japan are scarce. This prospective, observational, post-marketing Specified Use-Results Survey evaluated the real-world safety and effectiveness of romiplostim for 2 years. All patients treated with romiplostim during the survey period were eligible. Of the 1622 patients in the safety analysis set, 94.08% (1526/1622) had chronic ITP. The mean single dose of romiplostim was stable after 12 weeks and remained < 6 μg/kg in approximately 70% of patients until 104 weeks. Within 2 years, 14.92% of patients discontinued romiplostim because of adverse events, while 6.47% discontinued because of suspected adverse drug reactions. In contrast, 14.00% of patients discontinued romiplostim because of symptom improvement. Before romiplostim initiation, platelet count was < 2.0 × 10
4
/µL in 60.54% of patients, and the mean platelet count was 2.84 ± 5.76 × 10
4
/µL. Platelet count was 9.19 ± 13.01 × 10
4
/µL after 4 weeks, and remained between 10.34 ± 10.72 and 12.38 ± 12.63 × 10
4
/µL from 8 to 104 weeks of treatment. No specific concerns were revealed regarding the safety and effectiveness of romiplostim in chronic ITP; the findings demonstrated a favorable risk–benefit balance for romiplostim in this population.
Trial registration
: UMIN000047864 (
www.umin.ac.jp/ctr
).</abstract><cop>Singapore</cop><pub>Springer Nature Singapore</pub><pmid>39342544</pmid><doi>10.1007/s12185-024-03847-4</doi><tpages>10</tpages><orcidid>https://orcid.org/0000-0001-6563-3093</orcidid><oa>free_for_read</oa></addata></record> |
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| identifier | ISSN: 0925-5710 |
| ispartof | International journal of hematology, 2024-12, Vol.120 (6), p.665-674 |
| issn | 0925-5710 1865-3774 1865-3774 |
| language | eng |
| recordid | cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_11588964 |
| source | MEDLINE; SpringerNature Journals |
| subjects | Adolescent Adult Aged Aged, 80 and over Chronic Disease Effectiveness Female Hematological diseases Hematology Humans Idiopathic thrombocytopenic purpura Japan Male Marketing Medicine Medicine & Public Health Middle Aged Oncology Original Original Article Platelet Count Platelets Product Surveillance, Postmarketing Prospective Studies Purpura Purpura, Thrombocytopenic, Idiopathic - drug therapy Receptors, Fc - administration & dosage Receptors, Fc - therapeutic use Recombinant Fusion Proteins - administration & dosage Recombinant Fusion Proteins - adverse effects Recombinant Fusion Proteins - therapeutic use Safety Side effects Surveys Thrombocytopenic purpura Thrombopoietin - administration & dosage Thrombopoietin - adverse effects Thrombopoietin - therapeutic use Time Factors Treatment Outcome UMIN UMIN000047864 Young Adult |
| title | Long-term safety and effectiveness of romiplostim for chronic idiopathic thrombocytopenic purpura in real-world settings |
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