Results of a randomized, double‐blind study of romiplostim versus placebo in patients with low/intermediate‐1–risk myelodysplastic syndrome and thrombocytopenia
BACKGROUND Thrombocytopenia in patients with myelodysplastic syndrome (MDS) is associated with shortened survival and an increased risk of evolution to acute myeloid leukemia (AML). In this study, the authors evaluated the efficacy of romiplostim in patients who had thrombocytopenia with low‐risk/in...
Gespeichert in:
| Veröffentlicht in: | Cancer 2014-06, Vol.120 (12), p.1838-1846 |
|---|---|
| Hauptverfasser: | , , , , , , , , , , , , |
| Format: | Artikel |
| Sprache: | eng |
| Schlagworte: | |
| Online-Zugang: | Volltext |
| Tags: |
Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!
|
| container_end_page | 1846 |
|---|---|
| container_issue | 12 |
| container_start_page | 1838 |
| container_title | Cancer |
| container_volume | 120 |
| creator | Giagounidis, Aristoteles Mufti, Ghulam J. Fenaux, Pierre Sekeres, Mikkael A. Szer, Jeffrey Platzbecker, Uwe Kuendgen, Andrea Gaidano, Gianluca Wiktor‐Jedrzejczak, Wieslaw Hu, Kuolung Woodard, Paul Yang, Allen S. Kantarjian, Hagop M. |
| description | BACKGROUND
Thrombocytopenia in patients with myelodysplastic syndrome (MDS) is associated with shortened survival and an increased risk of evolution to acute myeloid leukemia (AML). In this study, the authors evaluated the efficacy of romiplostim in patients who had thrombocytopenia with low‐risk/intermediate‐1–risk MDS.
METHODS
Patients who had thrombocytopenia with low‐risk/intermediate‐1–risk MDS (N = 250) were randomized 2:1 to receive romiplostim or placebo weekly for 58 weeks.
RESULTS
The primary endpoint— the number of clinically significant bleeding events (CSBEs) per patient—had a hazard ratio for romiplostim:placebo of 0.83 (95% confidence interval, 0.66‐1.05; P = .13). CSBEs were reduced significantly in the romiplostim group for patients who had baseline platelet counts ≥20 × 109/L (P |
| doi_str_mv | 10.1002/cncr.28663 |
| format | Article |
| fullrecord | <record><control><sourceid>proquest_pubme</sourceid><recordid>TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4298760</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>1532951433</sourcerecordid><originalsourceid>FETCH-LOGICAL-c4503-192e6fcb23dcb15ae6e6507a8a5b09ff00779e62dfbd90087c2fb26b30f72b923</originalsourceid><addsrcrecordid>eNp9kcuKFDEYhYMoTju68QEkG0HEmsmlrhtBGm8wKAwK7kIuf9nRVFImVdOUq3kEwXfwweZJTNvtqBtXSciXc07-g9B9Sk4oIexUex1PWFvX_AZaUdI1BaElu4lWhJC2qEr-4QjdSelTPjas4rfRESsbUpdtt0I_ziHNbko49FjiKL0Jg_0K5gk2YVYOri6_KWe9wWmazbKjYgZGF9JkB3wBMc0Jj05qUAFbj0c5WfBZb2unDXZhe2r9BHEAY-W0U6NXl9-jTZ_xsIALZkn5cdbSOC3eZG3AOQOeNnmrgl6mMIK38i661UuX4N5hPUbvXzx_t35VnL19-Xr97KzQZUV4QTsGda8V40YrWkmooa5II1tZKdL1fR5A00HNTK9Ml4fTaNYrVitO-oapjvFj9HSvO84qZ9b5K1E6MUY7yLiIIK3498bbjfgYLkTJurapSRZ4dBCI4csMaRKDTRqckx7CnAStOOsqWnKe0cd7VMeQUoT-2oYSsStW7IoVv4rN8IO_g12jv5vMwMMDIJOWrs9Vapv-cG3FWEl2Aeme21oHy38sxfrN-nxv_hMlwcS9</addsrcrecordid><sourcetype>Open Access Repository</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>1532951433</pqid></control><display><type>article</type><title>Results of a randomized, double‐blind study of romiplostim versus placebo in patients with low/intermediate‐1–risk myelodysplastic syndrome and thrombocytopenia</title><source>MEDLINE</source><source>Wiley Online Library Journals Frontfile Complete</source><source>Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals</source><source>Wiley Free Content</source><source>Alma/SFX Local Collection</source><creator>Giagounidis, Aristoteles ; Mufti, Ghulam J. ; Fenaux, Pierre ; Sekeres, Mikkael A. ; Szer, Jeffrey ; Platzbecker, Uwe ; Kuendgen, Andrea ; Gaidano, Gianluca ; Wiktor‐Jedrzejczak, Wieslaw ; Hu, Kuolung ; Woodard, Paul ; Yang, Allen S. ; Kantarjian, Hagop M.</creator><creatorcontrib>Giagounidis, Aristoteles ; Mufti, Ghulam J. ; Fenaux, Pierre ; Sekeres, Mikkael A. ; Szer, Jeffrey ; Platzbecker, Uwe ; Kuendgen, Andrea ; Gaidano, Gianluca ; Wiktor‐Jedrzejczak, Wieslaw ; Hu, Kuolung ; Woodard, Paul ; Yang, Allen S. ; Kantarjian, Hagop M.</creatorcontrib><description>BACKGROUND
Thrombocytopenia in patients with myelodysplastic syndrome (MDS) is associated with shortened survival and an increased risk of evolution to acute myeloid leukemia (AML). In this study, the authors evaluated the efficacy of romiplostim in patients who had thrombocytopenia with low‐risk/intermediate‐1–risk MDS.
METHODS
Patients who had thrombocytopenia with low‐risk/intermediate‐1–risk MDS (N = 250) were randomized 2:1 to receive romiplostim or placebo weekly for 58 weeks.
RESULTS
The primary endpoint— the number of clinically significant bleeding events (CSBEs) per patient—had a hazard ratio for romiplostim:placebo of 0.83 (95% confidence interval, 0.66‐1.05; P = .13). CSBEs were reduced significantly in the romiplostim group for patients who had baseline platelet counts ≥20 × 109/L (P < .0001). For patients who had baseline platelet counts <20 × 109/L, there was no difference in the number of CSBEs, but the platelet transfusion rates were higher in the placebo group (P < .0001), which may have affected the overall CSBE results in this group with severe thrombocytopenia. The incidence of bleeding events was reduced significantly in the romiplostim group (relative risk, 0.92), as were protocol‐defined platelet transfusions (relative risk, 0.77). Platelet response rates according to 2006 International Working Group criteria were higher for the group that received romiplostim (odds ratio, 15.6). On the basis of interim data, an independent data monitoring committee advised halting study drug because of concerns regarding excess blasts and AML rates with romiplostim (interim hazard ratio, 2.51). At 58 weeks, the AML rates were 6% in the romiplostim group and 4.9% in the placebo group (hazard ratio, 1.20; 95% confidence interval, 0.38‐3.84), and the overall survival rates were similar.
CONCLUSIONS
Romiplostim treatment in patients with low‐risk/intermediate‐1–risk MDS increased platelet counts and decreased the number of bleeding events and platelet transfusions. Although study drug was discontinued because of an initial concern of AML risk, survival and AML rates were similar with romiplostim and placebo. Cancer 2014;120:1838–1846. © 2014 Amgen, Inc. Cancer published by Wiley Periodicals, Inc. on behalf of American Cancer Society.
Romiplostim increases platelet counts and decreases bleeding events and platelet transfusions in patients with low‐risk/intermediate‐1–risk myelodysplastic syndrome. The study drug has been stopped because of concerns regarding increased blast counts and progression to acute myeloid leukemia; survival and acute myeloid leukemia rates are similar, and follow‐up is ongoing.</description><identifier>ISSN: 0008-543X</identifier><identifier>EISSN: 1097-0142</identifier><identifier>DOI: 10.1002/cncr.28663</identifier><identifier>PMID: 24706489</identifier><identifier>CODEN: CANCAR</identifier><language>eng</language><publisher>Hoboken, NJ: Wiley-Blackwell</publisher><subject>Aged ; Biological and medical sciences ; Double-Blind Method ; drug therapy ; Female ; Hematologic and hematopoietic diseases ; Humans ; Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis ; Male ; Medical sciences ; Middle Aged ; myelodysplastic syndromes ; Myelodysplastic Syndromes - blood ; Myelodysplastic Syndromes - drug therapy ; Myelodysplastic Syndromes - pathology ; Original ; Placebos ; randomized controlled trial ; Receptors, Fc - therapeutic use ; Recombinant Fusion Proteins - therapeutic use ; romiplostim ; Survival Analysis ; thrombocytopenia ; Thrombocytopenia - drug therapy ; Thrombocytopenia - pathology ; Thrombopoietin - therapeutic use ; Treatment Outcome ; Tumors</subject><ispartof>Cancer, 2014-06, Vol.120 (12), p.1838-1846</ispartof><rights>2014 Amgen, Inc. Cancer published by Wiley Periodicals, Inc. on behalf of American Cancer Society.</rights><rights>2015 INIST-CNRS</rights><rights>2014 Amgen, Inc. Cancer published by Wiley Periodicals, Inc. on behalf of American Cancer Society. 2014</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c4503-192e6fcb23dcb15ae6e6507a8a5b09ff00779e62dfbd90087c2fb26b30f72b923</citedby><cites>FETCH-LOGICAL-c4503-192e6fcb23dcb15ae6e6507a8a5b09ff00779e62dfbd90087c2fb26b30f72b923</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://onlinelibrary.wiley.com/doi/pdf/10.1002%2Fcncr.28663$$EPDF$$P50$$Gwiley$$Hfree_for_read</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1002%2Fcncr.28663$$EHTML$$P50$$Gwiley$$Hfree_for_read</linktohtml><link.rule.ids>230,314,777,781,882,1412,1428,27905,27906,45555,45556,46390,46814</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=28522400$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/24706489$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Giagounidis, Aristoteles</creatorcontrib><creatorcontrib>Mufti, Ghulam J.</creatorcontrib><creatorcontrib>Fenaux, Pierre</creatorcontrib><creatorcontrib>Sekeres, Mikkael A.</creatorcontrib><creatorcontrib>Szer, Jeffrey</creatorcontrib><creatorcontrib>Platzbecker, Uwe</creatorcontrib><creatorcontrib>Kuendgen, Andrea</creatorcontrib><creatorcontrib>Gaidano, Gianluca</creatorcontrib><creatorcontrib>Wiktor‐Jedrzejczak, Wieslaw</creatorcontrib><creatorcontrib>Hu, Kuolung</creatorcontrib><creatorcontrib>Woodard, Paul</creatorcontrib><creatorcontrib>Yang, Allen S.</creatorcontrib><creatorcontrib>Kantarjian, Hagop M.</creatorcontrib><title>Results of a randomized, double‐blind study of romiplostim versus placebo in patients with low/intermediate‐1–risk myelodysplastic syndrome and thrombocytopenia</title><title>Cancer</title><addtitle>Cancer</addtitle><description>BACKGROUND
Thrombocytopenia in patients with myelodysplastic syndrome (MDS) is associated with shortened survival and an increased risk of evolution to acute myeloid leukemia (AML). In this study, the authors evaluated the efficacy of romiplostim in patients who had thrombocytopenia with low‐risk/intermediate‐1–risk MDS.
METHODS
Patients who had thrombocytopenia with low‐risk/intermediate‐1–risk MDS (N = 250) were randomized 2:1 to receive romiplostim or placebo weekly for 58 weeks.
RESULTS
The primary endpoint— the number of clinically significant bleeding events (CSBEs) per patient—had a hazard ratio for romiplostim:placebo of 0.83 (95% confidence interval, 0.66‐1.05; P = .13). CSBEs were reduced significantly in the romiplostim group for patients who had baseline platelet counts ≥20 × 109/L (P < .0001). For patients who had baseline platelet counts <20 × 109/L, there was no difference in the number of CSBEs, but the platelet transfusion rates were higher in the placebo group (P < .0001), which may have affected the overall CSBE results in this group with severe thrombocytopenia. The incidence of bleeding events was reduced significantly in the romiplostim group (relative risk, 0.92), as were protocol‐defined platelet transfusions (relative risk, 0.77). Platelet response rates according to 2006 International Working Group criteria were higher for the group that received romiplostim (odds ratio, 15.6). On the basis of interim data, an independent data monitoring committee advised halting study drug because of concerns regarding excess blasts and AML rates with romiplostim (interim hazard ratio, 2.51). At 58 weeks, the AML rates were 6% in the romiplostim group and 4.9% in the placebo group (hazard ratio, 1.20; 95% confidence interval, 0.38‐3.84), and the overall survival rates were similar.
CONCLUSIONS
Romiplostim treatment in patients with low‐risk/intermediate‐1–risk MDS increased platelet counts and decreased the number of bleeding events and platelet transfusions. Although study drug was discontinued because of an initial concern of AML risk, survival and AML rates were similar with romiplostim and placebo. Cancer 2014;120:1838–1846. © 2014 Amgen, Inc. Cancer published by Wiley Periodicals, Inc. on behalf of American Cancer Society.
Romiplostim increases platelet counts and decreases bleeding events and platelet transfusions in patients with low‐risk/intermediate‐1–risk myelodysplastic syndrome. The study drug has been stopped because of concerns regarding increased blast counts and progression to acute myeloid leukemia; survival and acute myeloid leukemia rates are similar, and follow‐up is ongoing.</description><subject>Aged</subject><subject>Biological and medical sciences</subject><subject>Double-Blind Method</subject><subject>drug therapy</subject><subject>Female</subject><subject>Hematologic and hematopoietic diseases</subject><subject>Humans</subject><subject>Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis</subject><subject>Male</subject><subject>Medical sciences</subject><subject>Middle Aged</subject><subject>myelodysplastic syndromes</subject><subject>Myelodysplastic Syndromes - blood</subject><subject>Myelodysplastic Syndromes - drug therapy</subject><subject>Myelodysplastic Syndromes - pathology</subject><subject>Original</subject><subject>Placebos</subject><subject>randomized controlled trial</subject><subject>Receptors, Fc - therapeutic use</subject><subject>Recombinant Fusion Proteins - therapeutic use</subject><subject>romiplostim</subject><subject>Survival Analysis</subject><subject>thrombocytopenia</subject><subject>Thrombocytopenia - drug therapy</subject><subject>Thrombocytopenia - pathology</subject><subject>Thrombopoietin - therapeutic use</subject><subject>Treatment Outcome</subject><subject>Tumors</subject><issn>0008-543X</issn><issn>1097-0142</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2014</creationdate><recordtype>article</recordtype><sourceid>24P</sourceid><sourceid>WIN</sourceid><sourceid>EIF</sourceid><recordid>eNp9kcuKFDEYhYMoTju68QEkG0HEmsmlrhtBGm8wKAwK7kIuf9nRVFImVdOUq3kEwXfwweZJTNvtqBtXSciXc07-g9B9Sk4oIexUex1PWFvX_AZaUdI1BaElu4lWhJC2qEr-4QjdSelTPjas4rfRESsbUpdtt0I_ziHNbko49FjiKL0Jg_0K5gk2YVYOri6_KWe9wWmazbKjYgZGF9JkB3wBMc0Jj05qUAFbj0c5WfBZb2unDXZhe2r9BHEAY-W0U6NXl9-jTZ_xsIALZkn5cdbSOC3eZG3AOQOeNnmrgl6mMIK38i661UuX4N5hPUbvXzx_t35VnL19-Xr97KzQZUV4QTsGda8V40YrWkmooa5II1tZKdL1fR5A00HNTK9Ml4fTaNYrVitO-oapjvFj9HSvO84qZ9b5K1E6MUY7yLiIIK3498bbjfgYLkTJurapSRZ4dBCI4csMaRKDTRqckx7CnAStOOsqWnKe0cd7VMeQUoT-2oYSsStW7IoVv4rN8IO_g12jv5vMwMMDIJOWrs9Vapv-cG3FWEl2Aeme21oHy38sxfrN-nxv_hMlwcS9</recordid><startdate>20140615</startdate><enddate>20140615</enddate><creator>Giagounidis, Aristoteles</creator><creator>Mufti, Ghulam J.</creator><creator>Fenaux, Pierre</creator><creator>Sekeres, Mikkael A.</creator><creator>Szer, Jeffrey</creator><creator>Platzbecker, Uwe</creator><creator>Kuendgen, Andrea</creator><creator>Gaidano, Gianluca</creator><creator>Wiktor‐Jedrzejczak, Wieslaw</creator><creator>Hu, Kuolung</creator><creator>Woodard, Paul</creator><creator>Yang, Allen S.</creator><creator>Kantarjian, Hagop M.</creator><general>Wiley-Blackwell</general><general>BlackWell Publishing Ltd</general><scope>24P</scope><scope>WIN</scope><scope>IQODW</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>7X8</scope><scope>5PM</scope></search><sort><creationdate>20140615</creationdate><title>Results of a randomized, double‐blind study of romiplostim versus placebo in patients with low/intermediate‐1–risk myelodysplastic syndrome and thrombocytopenia</title><author>Giagounidis, Aristoteles ; Mufti, Ghulam J. ; Fenaux, Pierre ; Sekeres, Mikkael A. ; Szer, Jeffrey ; Platzbecker, Uwe ; Kuendgen, Andrea ; Gaidano, Gianluca ; Wiktor‐Jedrzejczak, Wieslaw ; Hu, Kuolung ; Woodard, Paul ; Yang, Allen S. ; Kantarjian, Hagop M.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c4503-192e6fcb23dcb15ae6e6507a8a5b09ff00779e62dfbd90087c2fb26b30f72b923</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2014</creationdate><topic>Aged</topic><topic>Biological and medical sciences</topic><topic>Double-Blind Method</topic><topic>drug therapy</topic><topic>Female</topic><topic>Hematologic and hematopoietic diseases</topic><topic>Humans</topic><topic>Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis</topic><topic>Male</topic><topic>Medical sciences</topic><topic>Middle Aged</topic><topic>myelodysplastic syndromes</topic><topic>Myelodysplastic Syndromes - blood</topic><topic>Myelodysplastic Syndromes - drug therapy</topic><topic>Myelodysplastic Syndromes - pathology</topic><topic>Original</topic><topic>Placebos</topic><topic>randomized controlled trial</topic><topic>Receptors, Fc - therapeutic use</topic><topic>Recombinant Fusion Proteins - therapeutic use</topic><topic>romiplostim</topic><topic>Survival Analysis</topic><topic>thrombocytopenia</topic><topic>Thrombocytopenia - drug therapy</topic><topic>Thrombocytopenia - pathology</topic><topic>Thrombopoietin - therapeutic use</topic><topic>Treatment Outcome</topic><topic>Tumors</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Giagounidis, Aristoteles</creatorcontrib><creatorcontrib>Mufti, Ghulam J.</creatorcontrib><creatorcontrib>Fenaux, Pierre</creatorcontrib><creatorcontrib>Sekeres, Mikkael A.</creatorcontrib><creatorcontrib>Szer, Jeffrey</creatorcontrib><creatorcontrib>Platzbecker, Uwe</creatorcontrib><creatorcontrib>Kuendgen, Andrea</creatorcontrib><creatorcontrib>Gaidano, Gianluca</creatorcontrib><creatorcontrib>Wiktor‐Jedrzejczak, Wieslaw</creatorcontrib><creatorcontrib>Hu, Kuolung</creatorcontrib><creatorcontrib>Woodard, Paul</creatorcontrib><creatorcontrib>Yang, Allen S.</creatorcontrib><creatorcontrib>Kantarjian, Hagop M.</creatorcontrib><collection>Wiley Online Library Open Access</collection><collection>Wiley Free Content</collection><collection>Pascal-Francis</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Cancer</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Giagounidis, Aristoteles</au><au>Mufti, Ghulam J.</au><au>Fenaux, Pierre</au><au>Sekeres, Mikkael A.</au><au>Szer, Jeffrey</au><au>Platzbecker, Uwe</au><au>Kuendgen, Andrea</au><au>Gaidano, Gianluca</au><au>Wiktor‐Jedrzejczak, Wieslaw</au><au>Hu, Kuolung</au><au>Woodard, Paul</au><au>Yang, Allen S.</au><au>Kantarjian, Hagop M.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Results of a randomized, double‐blind study of romiplostim versus placebo in patients with low/intermediate‐1–risk myelodysplastic syndrome and thrombocytopenia</atitle><jtitle>Cancer</jtitle><addtitle>Cancer</addtitle><date>2014-06-15</date><risdate>2014</risdate><volume>120</volume><issue>12</issue><spage>1838</spage><epage>1846</epage><pages>1838-1846</pages><issn>0008-543X</issn><eissn>1097-0142</eissn><coden>CANCAR</coden><abstract>BACKGROUND
Thrombocytopenia in patients with myelodysplastic syndrome (MDS) is associated with shortened survival and an increased risk of evolution to acute myeloid leukemia (AML). In this study, the authors evaluated the efficacy of romiplostim in patients who had thrombocytopenia with low‐risk/intermediate‐1–risk MDS.
METHODS
Patients who had thrombocytopenia with low‐risk/intermediate‐1–risk MDS (N = 250) were randomized 2:1 to receive romiplostim or placebo weekly for 58 weeks.
RESULTS
The primary endpoint— the number of clinically significant bleeding events (CSBEs) per patient—had a hazard ratio for romiplostim:placebo of 0.83 (95% confidence interval, 0.66‐1.05; P = .13). CSBEs were reduced significantly in the romiplostim group for patients who had baseline platelet counts ≥20 × 109/L (P < .0001). For patients who had baseline platelet counts <20 × 109/L, there was no difference in the number of CSBEs, but the platelet transfusion rates were higher in the placebo group (P < .0001), which may have affected the overall CSBE results in this group with severe thrombocytopenia. The incidence of bleeding events was reduced significantly in the romiplostim group (relative risk, 0.92), as were protocol‐defined platelet transfusions (relative risk, 0.77). Platelet response rates according to 2006 International Working Group criteria were higher for the group that received romiplostim (odds ratio, 15.6). On the basis of interim data, an independent data monitoring committee advised halting study drug because of concerns regarding excess blasts and AML rates with romiplostim (interim hazard ratio, 2.51). At 58 weeks, the AML rates were 6% in the romiplostim group and 4.9% in the placebo group (hazard ratio, 1.20; 95% confidence interval, 0.38‐3.84), and the overall survival rates were similar.
CONCLUSIONS
Romiplostim treatment in patients with low‐risk/intermediate‐1–risk MDS increased platelet counts and decreased the number of bleeding events and platelet transfusions. Although study drug was discontinued because of an initial concern of AML risk, survival and AML rates were similar with romiplostim and placebo. Cancer 2014;120:1838–1846. © 2014 Amgen, Inc. Cancer published by Wiley Periodicals, Inc. on behalf of American Cancer Society.
Romiplostim increases platelet counts and decreases bleeding events and platelet transfusions in patients with low‐risk/intermediate‐1–risk myelodysplastic syndrome. The study drug has been stopped because of concerns regarding increased blast counts and progression to acute myeloid leukemia; survival and acute myeloid leukemia rates are similar, and follow‐up is ongoing.</abstract><cop>Hoboken, NJ</cop><pub>Wiley-Blackwell</pub><pmid>24706489</pmid><doi>10.1002/cncr.28663</doi><tpages>9</tpages><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0008-543X |
| ispartof | Cancer, 2014-06, Vol.120 (12), p.1838-1846 |
| issn | 0008-543X 1097-0142 |
| language | eng |
| recordid | cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4298760 |
| source | MEDLINE; Wiley Online Library Journals Frontfile Complete; Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals; Wiley Free Content; Alma/SFX Local Collection |
| subjects | Aged Biological and medical sciences Double-Blind Method drug therapy Female Hematologic and hematopoietic diseases Humans Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis Male Medical sciences Middle Aged myelodysplastic syndromes Myelodysplastic Syndromes - blood Myelodysplastic Syndromes - drug therapy Myelodysplastic Syndromes - pathology Original Placebos randomized controlled trial Receptors, Fc - therapeutic use Recombinant Fusion Proteins - therapeutic use romiplostim Survival Analysis thrombocytopenia Thrombocytopenia - drug therapy Thrombocytopenia - pathology Thrombopoietin - therapeutic use Treatment Outcome Tumors |
| title | Results of a randomized, double‐blind study of romiplostim versus placebo in patients with low/intermediate‐1–risk myelodysplastic syndrome and thrombocytopenia |
| url | https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2025-01-20T15%3A43%3A19IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-proquest_pubme&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.genre=article&rft.atitle=Results%20of%20a%20randomized,%20double%E2%80%90blind%20study%20of%20romiplostim%20versus%20placebo%20in%20patients%20with%20low/intermediate%E2%80%901%E2%80%93risk%20myelodysplastic%20syndrome%20and%20thrombocytopenia&rft.jtitle=Cancer&rft.au=Giagounidis,%20Aristoteles&rft.date=2014-06-15&rft.volume=120&rft.issue=12&rft.spage=1838&rft.epage=1846&rft.pages=1838-1846&rft.issn=0008-543X&rft.eissn=1097-0142&rft.coden=CANCAR&rft_id=info:doi/10.1002/cncr.28663&rft_dat=%3Cproquest_pubme%3E1532951433%3C/proquest_pubme%3E%3Curl%3E%3C/url%3E&disable_directlink=true&sfx.directlink=off&sfx.report_link=0&rft_id=info:oai/&rft_pqid=1532951433&rft_id=info:pmid/24706489&rfr_iscdi=true |