Efficacy and safety of treatments in newly diagnosed adult primary immune thrombocytopenia: a systematic review and network meta-analysisResearch in context
Summary: Background: Immune thrombocytopenia is an autoimmune disease characterised by decreased platelet count. In recent years, novel therapeutic regimens have been investigated in randomised controlled trials (RCTs). We aimed to compare the efficacy and safety of different treatments in newly di...
| Main Authors: | , , , , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2023-02-01
|
| Series: | EClinicalMedicine |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2589537022005065 |
| _version_ | 1797895573634809856 |
|---|---|
| author | Yun Wang Lei Sheng Fengjiao Han Qiuyu Guo Zihan Zhang Yu Hou Qi Feng Hai Zhou Xuebin Ji Jun Peng Ming Hou Miao Xu |
| author_facet | Yun Wang Lei Sheng Fengjiao Han Qiuyu Guo Zihan Zhang Yu Hou Qi Feng Hai Zhou Xuebin Ji Jun Peng Ming Hou Miao Xu |
| author_sort | Yun Wang |
| collection | DOAJ |
| description | Summary: Background: Immune thrombocytopenia is an autoimmune disease characterised by decreased platelet count. In recent years, novel therapeutic regimens have been investigated in randomised controlled trials (RCTs). We aimed to compare the efficacy and safety of different treatments in newly diagnosed adult primary immune thrombocytopenia. Methods: We did a systematic review and network meta-analysis of RCTs involving treatments for newly diagnosed primary immune thrombocytopenia. PubMed, Embase, the Cochrane Central Register of Controlled Trials, and ClinicalTrials.gov databases were searched up to April 31, 2022. The primary outcomes were 6-month sustained response and early response. Secondary outcome was grade 3 or higher adverse events. This study is registered with PROSPERO (CRD42022296179). Findings: Eighteen RCTs (n = 1944) were included in this study. Pairwise meta-analysis showed that the percentage of patients achieving early response was higher in the dexamethasone-containing doublet group than in the dexamethasone group (79.7% vs 68.7%, odds ratio [OR] 1.82, 95% CI 1.10–3.02). The difference was more profound for sustained response (60.5% vs 37.4%, OR 2.57, 95% CI 1.95–3.40). Network meta-analysis showed that dexamethasone plus recombinant human thrombopoietin ranked first for early response, followed by dexamethasone plus oseltamivir or tacrolimus. Rituximab plus prednisolone achieved highest sustained response, followed by dexamethasone plus all-trans retinoic acid or rituximab. Rituximab plus dexamethasone showed 15.3% of grade 3 or higher adverse events, followed by prednis(ol)one (4.8%) and all-trans retinoic acid plus dexamethasone (4.7%). Interpretation: Our findings suggested that compared with monotherapy dexamethasone or prednis(ol)one, the combined regimens had better early and sustained responses. rhTPO plus dexamethasone ranked top in early response, while rituximab plus corticosteroids obtained the best sustained response, but with more adverse events. Adding oseltamivir, all-trans retinoic acid or tacrolimus to dexamethasone reached equally encouraging sustained response, without compromising safety profile. Although this network meta-analysis compared all the therapeutic regimens up to date, more head-to-head RCTs with larger sample size are warranted to make direct comparison among these strategies. Funding: National Natural Science Foundation of China, Major Research Plan of National Natural Science Foundation of China, Shandong Provincial Natural Science Foundation and Young Taishan Scholar Foundation of Shandong Province. |
| first_indexed | 2024-04-10T07:28:00Z |
| format | Article |
| id | doaj.art-cd4fdb4361ce4523acde3446e3257313 |
| institution | Directory Open Access Journal |
| issn | 2589-5370 |
| language | English |
| last_indexed | 2024-04-10T07:28:00Z |
| publishDate | 2023-02-01 |
| publisher | Elsevier |
| record_format | Article |
| series | EClinicalMedicine |
| spelling | doaj.art-cd4fdb4361ce4523acde3446e32573132023-02-24T04:31:12ZengElsevierEClinicalMedicine2589-53702023-02-0156101777Efficacy and safety of treatments in newly diagnosed adult primary immune thrombocytopenia: a systematic review and network meta-analysisResearch in contextYun Wang0Lei Sheng1Fengjiao Han2Qiuyu Guo3Zihan Zhang4Yu Hou5Qi Feng6Hai Zhou7Xuebin Ji8Jun Peng9Ming Hou10Miao Xu11Department of Hematology, Cheeloo College of Medicine, Qilu Hospital of Shandong University, Jinan, ChinaDepartment of General Surgery, Cheeloo College of Medicine, Qilu Hospital of Shandong University, Jinan, ChinaDepartment of Hematology, Cheeloo College of Medicine, Qilu Hospital of Shandong University, Jinan, ChinaDepartment of Hematology, Cheeloo College of Medicine, Qilu Hospital of Shandong University, Jinan, ChinaDepartment of Hematology, Cheeloo College of Medicine, Qilu Hospital of Shandong University, Jinan, ChinaDepartment of Hematology, Cheeloo College of Medicine, Qilu Hospital of Shandong University, Jinan, ChinaDepartment of Hematology, Cheeloo College of Medicine, Qilu Hospital of Shandong University, Jinan, ChinaDepartment of Hematology, Cheeloo College of Medicine, Qilu Hospital of Shandong University, Jinan, ChinaDepartment of Hematology, Cheeloo College of Medicine, Qilu Hospital of Shandong University, Jinan, ChinaDepartment of Hematology, Cheeloo College of Medicine, Qilu Hospital of Shandong University, Jinan, China; Advanced Medical Research Institute, Shandong University, Jinan, ChinaDepartment of Hematology, Cheeloo College of Medicine, Qilu Hospital of Shandong University, Jinan, China; Corresponding author. Department of Hematology, Cheeloo College of Medicine, Qilu Hospital of Shandong University, Wenhuaxi Road, Jinan, China.Department of Hematology, Cheeloo College of Medicine, Qilu Hospital of Shandong University, Jinan, China; Corresponding author. Department of Hematology, Cheeloo College of Medicine, Qilu Hospital of Shandong University, Wenhuaxi Road, Jinan, China.Summary: Background: Immune thrombocytopenia is an autoimmune disease characterised by decreased platelet count. In recent years, novel therapeutic regimens have been investigated in randomised controlled trials (RCTs). We aimed to compare the efficacy and safety of different treatments in newly diagnosed adult primary immune thrombocytopenia. Methods: We did a systematic review and network meta-analysis of RCTs involving treatments for newly diagnosed primary immune thrombocytopenia. PubMed, Embase, the Cochrane Central Register of Controlled Trials, and ClinicalTrials.gov databases were searched up to April 31, 2022. The primary outcomes were 6-month sustained response and early response. Secondary outcome was grade 3 or higher adverse events. This study is registered with PROSPERO (CRD42022296179). Findings: Eighteen RCTs (n = 1944) were included in this study. Pairwise meta-analysis showed that the percentage of patients achieving early response was higher in the dexamethasone-containing doublet group than in the dexamethasone group (79.7% vs 68.7%, odds ratio [OR] 1.82, 95% CI 1.10–3.02). The difference was more profound for sustained response (60.5% vs 37.4%, OR 2.57, 95% CI 1.95–3.40). Network meta-analysis showed that dexamethasone plus recombinant human thrombopoietin ranked first for early response, followed by dexamethasone plus oseltamivir or tacrolimus. Rituximab plus prednisolone achieved highest sustained response, followed by dexamethasone plus all-trans retinoic acid or rituximab. Rituximab plus dexamethasone showed 15.3% of grade 3 or higher adverse events, followed by prednis(ol)one (4.8%) and all-trans retinoic acid plus dexamethasone (4.7%). Interpretation: Our findings suggested that compared with monotherapy dexamethasone or prednis(ol)one, the combined regimens had better early and sustained responses. rhTPO plus dexamethasone ranked top in early response, while rituximab plus corticosteroids obtained the best sustained response, but with more adverse events. Adding oseltamivir, all-trans retinoic acid or tacrolimus to dexamethasone reached equally encouraging sustained response, without compromising safety profile. Although this network meta-analysis compared all the therapeutic regimens up to date, more head-to-head RCTs with larger sample size are warranted to make direct comparison among these strategies. Funding: National Natural Science Foundation of China, Major Research Plan of National Natural Science Foundation of China, Shandong Provincial Natural Science Foundation and Young Taishan Scholar Foundation of Shandong Province.http://www.sciencedirect.com/science/article/pii/S2589537022005065Primary immune thrombocytopeniaNetwork meta-analysisRandomized controlled trialsDexamethasonePrednis(ol)oneRituximab |
| spellingShingle | Yun Wang Lei Sheng Fengjiao Han Qiuyu Guo Zihan Zhang Yu Hou Qi Feng Hai Zhou Xuebin Ji Jun Peng Ming Hou Miao Xu Efficacy and safety of treatments in newly diagnosed adult primary immune thrombocytopenia: a systematic review and network meta-analysisResearch in context EClinicalMedicine Primary immune thrombocytopenia Network meta-analysis Randomized controlled trials Dexamethasone Prednis(ol)one Rituximab |
| title | Efficacy and safety of treatments in newly diagnosed adult primary immune thrombocytopenia: a systematic review and network meta-analysisResearch in context |
| title_full | Efficacy and safety of treatments in newly diagnosed adult primary immune thrombocytopenia: a systematic review and network meta-analysisResearch in context |
| title_fullStr | Efficacy and safety of treatments in newly diagnosed adult primary immune thrombocytopenia: a systematic review and network meta-analysisResearch in context |
| title_full_unstemmed | Efficacy and safety of treatments in newly diagnosed adult primary immune thrombocytopenia: a systematic review and network meta-analysisResearch in context |
| title_short | Efficacy and safety of treatments in newly diagnosed adult primary immune thrombocytopenia: a systematic review and network meta-analysisResearch in context |
| title_sort | efficacy and safety of treatments in newly diagnosed adult primary immune thrombocytopenia a systematic review and network meta analysisresearch in context |
| topic | Primary immune thrombocytopenia Network meta-analysis Randomized controlled trials Dexamethasone Prednis(ol)one Rituximab |
| url | http://www.sciencedirect.com/science/article/pii/S2589537022005065 |
| work_keys_str_mv | AT yunwang efficacyandsafetyoftreatmentsinnewlydiagnosedadultprimaryimmunethrombocytopeniaasystematicreviewandnetworkmetaanalysisresearchincontext AT leisheng efficacyandsafetyoftreatmentsinnewlydiagnosedadultprimaryimmunethrombocytopeniaasystematicreviewandnetworkmetaanalysisresearchincontext AT fengjiaohan efficacyandsafetyoftreatmentsinnewlydiagnosedadultprimaryimmunethrombocytopeniaasystematicreviewandnetworkmetaanalysisresearchincontext AT qiuyuguo efficacyandsafetyoftreatmentsinnewlydiagnosedadultprimaryimmunethrombocytopeniaasystematicreviewandnetworkmetaanalysisresearchincontext AT zihanzhang efficacyandsafetyoftreatmentsinnewlydiagnosedadultprimaryimmunethrombocytopeniaasystematicreviewandnetworkmetaanalysisresearchincontext AT yuhou efficacyandsafetyoftreatmentsinnewlydiagnosedadultprimaryimmunethrombocytopeniaasystematicreviewandnetworkmetaanalysisresearchincontext AT qifeng efficacyandsafetyoftreatmentsinnewlydiagnosedadultprimaryimmunethrombocytopeniaasystematicreviewandnetworkmetaanalysisresearchincontext AT haizhou efficacyandsafetyoftreatmentsinnewlydiagnosedadultprimaryimmunethrombocytopeniaasystematicreviewandnetworkmetaanalysisresearchincontext AT xuebinji efficacyandsafetyoftreatmentsinnewlydiagnosedadultprimaryimmunethrombocytopeniaasystematicreviewandnetworkmetaanalysisresearchincontext AT junpeng efficacyandsafetyoftreatmentsinnewlydiagnosedadultprimaryimmunethrombocytopeniaasystematicreviewandnetworkmetaanalysisresearchincontext AT minghou efficacyandsafetyoftreatmentsinnewlydiagnosedadultprimaryimmunethrombocytopeniaasystematicreviewandnetworkmetaanalysisresearchincontext AT miaoxu efficacyandsafetyoftreatmentsinnewlydiagnosedadultprimaryimmunethrombocytopeniaasystematicreviewandnetworkmetaanalysisresearchincontext |