Efficacy and Safety of Thalidomide in Patients With Transfusion-Dependent Thalassemia
Objective To assess the efficacy and safety of thalidomide in children with transfusion-dependent thalassemia. Methods This prospective, single center, open-label study enrolled children aged 12–18 years, and who received thalidomide for a duration of 6 months at a starting dose of 2–3 mg/kg/day. Ef...
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| Veröffentlicht in: | Indian pediatrics 2021-07, Vol.58 (7), p.611-616 |
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| creator | Chandra, Jagdish Parakh, Nupur Sidharth Singh, Neha Sharma, Sunita Goel, Manish Pemde, Harish |
| description | Objective
To assess the efficacy and safety of thalidomide in children with transfusion-dependent thalassemia.
Methods
This prospective, single center, open-label study enrolled children aged 12–18 years, and who received thalidomide for a duration of 6 months at a starting dose of 2–3 mg/kg/day. Efficacy was assessed by reduction in transfusion requirement and rate of fall of hemoglobin. Efficacy was classified as major, moderate and minimal/no response depending on the reduction in transfusion requirement. Safety was assessed by adverse effects related to thalidomide.
Results
37 children [mean (SD) age, 14.7 (1.8) years were included. Rate of fall of hemoglobin reduced from a mean of 1.0 (0.24) g/week pre-thalidomide therapy to 0.58 (0.26) g/week after 6 months of thalidomide (
P |
| doi_str_mv | 10.1007/s13312-021-2254-y |
| format | Article |
| fullrecord | <record><control><sourceid>proquest_cross</sourceid><recordid>TN_cdi_proquest_miscellaneous_2555965127</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>2555965127</sourcerecordid><originalsourceid>FETCH-LOGICAL-c321t-dc424f2d70d0cdd554ec62020ced0ff59eccc63afb616b8485ff5c1c49f2d9653</originalsourceid><addsrcrecordid>eNp9kD1PwzAQhi0EEqXwA9g8shhsJ3aaEZXyIVUCiVaMlusP6ipxii8Z8u8xhJnpTnfvc9I9CF0zessore6AFQXjhHJGOBclGU_QjNZVSSoh6tPcU1YTSSU7RxcAB0p5wQWboe3K-2C0GbGOFr9r7_oRdx5v9roJtmuDdThE_Kb74GIP-CP0e7xJOoIfIHSRPLijizbvfhEN4NqgL9GZ1w24q786R9vH1Wb5TNavTy_L-zUxBWc9sabkpee2opYaa4UonZGccmqcpd6L2hljZKH9TjK5W5QLkYeGmbLOUC1FMUc3091j6r4GB71qAxjXNDq6bgDFRf5eCsarHGVT1KQOIDmvjim0Oo2KUfWjUE0KVVaofhSqMTN8YiBn46dL6tANKeaP_oG-Ab_zdZY</addsrcrecordid><sourcetype>Aggregation Database</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>2555965127</pqid></control><display><type>article</type><title>Efficacy and Safety of Thalidomide in Patients With Transfusion-Dependent Thalassemia</title><source>SpringerNature Journals</source><source>EZB-FREE-00999 freely available EZB journals</source><source>Alma/SFX Local Collection</source><creator>Chandra, Jagdish ; Parakh, Nupur ; Sidharth ; Singh, Neha ; Sharma, Sunita ; Goel, Manish ; Pemde, Harish</creator><creatorcontrib>Chandra, Jagdish ; Parakh, Nupur ; Sidharth ; Singh, Neha ; Sharma, Sunita ; Goel, Manish ; Pemde, Harish</creatorcontrib><description>Objective
To assess the efficacy and safety of thalidomide in children with transfusion-dependent thalassemia.
Methods
This prospective, single center, open-label study enrolled children aged 12–18 years, and who received thalidomide for a duration of 6 months at a starting dose of 2–3 mg/kg/day. Efficacy was assessed by reduction in transfusion requirement and rate of fall of hemoglobin. Efficacy was classified as major, moderate and minimal/no response depending on the reduction in transfusion requirement. Safety was assessed by adverse effects related to thalidomide.
Results
37 children [mean (SD) age, 14.7 (1.8) years were included. Rate of fall of hemoglobin reduced from a mean of 1.0 (0.24) g/week pre-thalidomide therapy to 0.58 (0.26) g/week after 6 months of thalidomide (
P
<0.001). 19 children (51.3%) had major response and 12 (32.4%) had moderate response. In 13.5% and 32.4% children response was observed within the first and second month of therapy, respectively. 15 (40.5%) children remained transfusion — free for a median (IQR) time of 6 (3–10) weeks of thalidomide therapy. Mean serum ferritin (SD) decreased from 1758.9 (835.1) to 1549.6(1016.9) (
P
<0.001). Mean HbF (SD) showed an increase from 2.95(2.6) to 49.2(33.3) (
P
<0.001). In 32 children, 47 adverse events were observed. Common adverse events were constipation and neutropenia (mostly mild).
Conclusions
Thalidomide resulted in major/moderate response in majority of children with transfusion-dependent thalassemia with satisfactory adverse effect profile.</description><identifier>ISSN: 0019-6061</identifier><identifier>EISSN: 0974-7559</identifier><identifier>DOI: 10.1007/s13312-021-2254-y</identifier><language>eng</language><publisher>New Delhi: Springer India</publisher><subject>Maternal and Child Health ; Medicine ; Medicine & Public Health ; Pediatric Surgery ; Pediatrics ; Research Paper</subject><ispartof>Indian pediatrics, 2021-07, Vol.58 (7), p.611-616</ispartof><rights>Indian Academy of Pediatrics 2021</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c321t-dc424f2d70d0cdd554ec62020ced0ff59eccc63afb616b8485ff5c1c49f2d9653</citedby><cites>FETCH-LOGICAL-c321t-dc424f2d70d0cdd554ec62020ced0ff59eccc63afb616b8485ff5c1c49f2d9653</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://link.springer.com/content/pdf/10.1007/s13312-021-2254-y$$EPDF$$P50$$Gspringer$$H</linktopdf><linktohtml>$$Uhttps://link.springer.com/10.1007/s13312-021-2254-y$$EHTML$$P50$$Gspringer$$H</linktohtml><link.rule.ids>314,780,784,27924,27925,41488,42557,51319</link.rule.ids></links><search><creatorcontrib>Chandra, Jagdish</creatorcontrib><creatorcontrib>Parakh, Nupur</creatorcontrib><creatorcontrib>Sidharth</creatorcontrib><creatorcontrib>Singh, Neha</creatorcontrib><creatorcontrib>Sharma, Sunita</creatorcontrib><creatorcontrib>Goel, Manish</creatorcontrib><creatorcontrib>Pemde, Harish</creatorcontrib><title>Efficacy and Safety of Thalidomide in Patients With Transfusion-Dependent Thalassemia</title><title>Indian pediatrics</title><addtitle>Indian Pediatr</addtitle><description>Objective
To assess the efficacy and safety of thalidomide in children with transfusion-dependent thalassemia.
Methods
This prospective, single center, open-label study enrolled children aged 12–18 years, and who received thalidomide for a duration of 6 months at a starting dose of 2–3 mg/kg/day. Efficacy was assessed by reduction in transfusion requirement and rate of fall of hemoglobin. Efficacy was classified as major, moderate and minimal/no response depending on the reduction in transfusion requirement. Safety was assessed by adverse effects related to thalidomide.
Results
37 children [mean (SD) age, 14.7 (1.8) years were included. Rate of fall of hemoglobin reduced from a mean of 1.0 (0.24) g/week pre-thalidomide therapy to 0.58 (0.26) g/week after 6 months of thalidomide (
P
<0.001). 19 children (51.3%) had major response and 12 (32.4%) had moderate response. In 13.5% and 32.4% children response was observed within the first and second month of therapy, respectively. 15 (40.5%) children remained transfusion — free for a median (IQR) time of 6 (3–10) weeks of thalidomide therapy. Mean serum ferritin (SD) decreased from 1758.9 (835.1) to 1549.6(1016.9) (
P
<0.001). Mean HbF (SD) showed an increase from 2.95(2.6) to 49.2(33.3) (
P
<0.001). In 32 children, 47 adverse events were observed. Common adverse events were constipation and neutropenia (mostly mild).
Conclusions
Thalidomide resulted in major/moderate response in majority of children with transfusion-dependent thalassemia with satisfactory adverse effect profile.</description><subject>Maternal and Child Health</subject><subject>Medicine</subject><subject>Medicine & Public Health</subject><subject>Pediatric Surgery</subject><subject>Pediatrics</subject><subject>Research Paper</subject><issn>0019-6061</issn><issn>0974-7559</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2021</creationdate><recordtype>article</recordtype><recordid>eNp9kD1PwzAQhi0EEqXwA9g8shhsJ3aaEZXyIVUCiVaMlusP6ipxii8Z8u8xhJnpTnfvc9I9CF0zessore6AFQXjhHJGOBclGU_QjNZVSSoh6tPcU1YTSSU7RxcAB0p5wQWboe3K-2C0GbGOFr9r7_oRdx5v9roJtmuDdThE_Kb74GIP-CP0e7xJOoIfIHSRPLijizbvfhEN4NqgL9GZ1w24q786R9vH1Wb5TNavTy_L-zUxBWc9sabkpee2opYaa4UonZGccmqcpd6L2hljZKH9TjK5W5QLkYeGmbLOUC1FMUc3091j6r4GB71qAxjXNDq6bgDFRf5eCsarHGVT1KQOIDmvjim0Oo2KUfWjUE0KVVaofhSqMTN8YiBn46dL6tANKeaP_oG-Ab_zdZY</recordid><startdate>20210701</startdate><enddate>20210701</enddate><creator>Chandra, Jagdish</creator><creator>Parakh, Nupur</creator><creator>Sidharth</creator><creator>Singh, Neha</creator><creator>Sharma, Sunita</creator><creator>Goel, Manish</creator><creator>Pemde, Harish</creator><general>Springer India</general><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope></search><sort><creationdate>20210701</creationdate><title>Efficacy and Safety of Thalidomide in Patients With Transfusion-Dependent Thalassemia</title><author>Chandra, Jagdish ; Parakh, Nupur ; Sidharth ; Singh, Neha ; Sharma, Sunita ; Goel, Manish ; Pemde, Harish</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c321t-dc424f2d70d0cdd554ec62020ced0ff59eccc63afb616b8485ff5c1c49f2d9653</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2021</creationdate><topic>Maternal and Child Health</topic><topic>Medicine</topic><topic>Medicine & Public Health</topic><topic>Pediatric Surgery</topic><topic>Pediatrics</topic><topic>Research Paper</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Chandra, Jagdish</creatorcontrib><creatorcontrib>Parakh, Nupur</creatorcontrib><creatorcontrib>Sidharth</creatorcontrib><creatorcontrib>Singh, Neha</creatorcontrib><creatorcontrib>Sharma, Sunita</creatorcontrib><creatorcontrib>Goel, Manish</creatorcontrib><creatorcontrib>Pemde, Harish</creatorcontrib><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><jtitle>Indian pediatrics</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Chandra, Jagdish</au><au>Parakh, Nupur</au><au>Sidharth</au><au>Singh, Neha</au><au>Sharma, Sunita</au><au>Goel, Manish</au><au>Pemde, Harish</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Efficacy and Safety of Thalidomide in Patients With Transfusion-Dependent Thalassemia</atitle><jtitle>Indian pediatrics</jtitle><stitle>Indian Pediatr</stitle><date>2021-07-01</date><risdate>2021</risdate><volume>58</volume><issue>7</issue><spage>611</spage><epage>616</epage><pages>611-616</pages><issn>0019-6061</issn><eissn>0974-7559</eissn><abstract>Objective
To assess the efficacy and safety of thalidomide in children with transfusion-dependent thalassemia.
Methods
This prospective, single center, open-label study enrolled children aged 12–18 years, and who received thalidomide for a duration of 6 months at a starting dose of 2–3 mg/kg/day. Efficacy was assessed by reduction in transfusion requirement and rate of fall of hemoglobin. Efficacy was classified as major, moderate and minimal/no response depending on the reduction in transfusion requirement. Safety was assessed by adverse effects related to thalidomide.
Results
37 children [mean (SD) age, 14.7 (1.8) years were included. Rate of fall of hemoglobin reduced from a mean of 1.0 (0.24) g/week pre-thalidomide therapy to 0.58 (0.26) g/week after 6 months of thalidomide (
P
<0.001). 19 children (51.3%) had major response and 12 (32.4%) had moderate response. In 13.5% and 32.4% children response was observed within the first and second month of therapy, respectively. 15 (40.5%) children remained transfusion — free for a median (IQR) time of 6 (3–10) weeks of thalidomide therapy. Mean serum ferritin (SD) decreased from 1758.9 (835.1) to 1549.6(1016.9) (
P
<0.001). Mean HbF (SD) showed an increase from 2.95(2.6) to 49.2(33.3) (
P
<0.001). In 32 children, 47 adverse events were observed. Common adverse events were constipation and neutropenia (mostly mild).
Conclusions
Thalidomide resulted in major/moderate response in majority of children with transfusion-dependent thalassemia with satisfactory adverse effect profile.</abstract><cop>New Delhi</cop><pub>Springer India</pub><doi>10.1007/s13312-021-2254-y</doi><tpages>6</tpages></addata></record> |
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| source | SpringerNature Journals; EZB-FREE-00999 freely available EZB journals; Alma/SFX Local Collection |
| subjects | Maternal and Child Health Medicine Medicine & Public Health Pediatric Surgery Pediatrics Research Paper |
| title | Efficacy and Safety of Thalidomide in Patients With Transfusion-Dependent Thalassemia |
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