4CPS-072 Real-world experience in haemophilia B patients after switching to fix extended half-life using pharmacokinetic population software and monocompartmental model

Background and ImportanceNew strategies have been developed for the prophylactic treatment of patients with haemophilia B (HB) such as extended half-life recombinant factor IX concentrates (rFIX EHL). These products have shown favourable pharmacokinetic properties, attaining a half-life 3- to 5-fold...

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Veröffentlicht in:European journal of hospital pharmacy. Science and practice 2023-03, Vol.30 (Suppl 1), p.A47-A48
Hauptverfasser: Juárez-Giménez, JC, Benítez-Hidalgo, O, Romero-Garrido, JA, Mateos-Salillas, C, González-Piñeiro, S, Montoro-Ronsano, JB
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container_end_page A48
container_issue Suppl 1
container_start_page A47
container_title European journal of hospital pharmacy. Science and practice
container_volume 30
creator Juárez-Giménez, JC
Benítez-Hidalgo, O
Romero-Garrido, JA
Mateos-Salillas, C
González-Piñeiro, S
Montoro-Ronsano, JB
description Background and ImportanceNew strategies have been developed for the prophylactic treatment of patients with haemophilia B (HB) such as extended half-life recombinant factor IX concentrates (rFIX EHL). These products have shown favourable pharmacokinetic properties, attaining a half-life 3- to 5-fold longer in rFIX EHL compared to standard FIX concentratesAim and ObjectivesEfficiency of a pharmacokinetic-based tailored prophylaxis-dosing schedule versus standard dosing (DS) is compared, in HB, treated with two rFIX-EHL.Material and MethodsObservational, analytical, prospective, multicentre study, involving HB patients, being treated with rFIX-EHL linked to albumin (rFIX-FP) or to fragment crystallisable (rFIX-Fc). Demographic and clinical data, and DS and dosing interval (DI) and actual FIX trough levels were recorded. Pharmacokinetic characterisation was performed following both a population (WAPPS-HEMO) and a linear one-compartment (OC) approach. For each approach and rFIX preparation, an estimation of the time to the target trough (5 IU FIX/dL) was made. Statistical analysis was performed by means of the Student-Fisher t-test.ResultsFifteen patients were included, nine being treated with rFIX-FP (mean age, 33 years; weight 60 kg), and six with rFIX-Fc (49 years, 86 kg). Mean DS was 3222 UI (SD, 1716) every 11,9 days (SD, 4,4) for rFIX-FP patients; and 4333 UI (SD, 606) every 14,0 days (SD, 0,0) for rFIX-Fc patients. The individual tailored DI, for a 0,05 UI/dL trough target was: applying OC; 13,6 days (SD, 5,1), -1,8 days (SD, 5,9) vs DS, representing 240 IU/day (SD, 136,1) for rFIX-FP (p=0,40), and 8,6 days (SD, 1,2), +5,4 days (SD, 1,2) vs DS, representing 508 IU/day (SD, 65,6), for rFIX-Fc, (p
doi_str_mv 10.1136/ejhpharm-2023-eahp.100
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These products have shown favourable pharmacokinetic properties, attaining a half-life 3- to 5-fold longer in rFIX EHL compared to standard FIX concentratesAim and ObjectivesEfficiency of a pharmacokinetic-based tailored prophylaxis-dosing schedule versus standard dosing (DS) is compared, in HB, treated with two rFIX-EHL.Material and MethodsObservational, analytical, prospective, multicentre study, involving HB patients, being treated with rFIX-EHL linked to albumin (rFIX-FP) or to fragment crystallisable (rFIX-Fc). Demographic and clinical data, and DS and dosing interval (DI) and actual FIX trough levels were recorded. Pharmacokinetic characterisation was performed following both a population (WAPPS-HEMO) and a linear one-compartment (OC) approach. For each approach and rFIX preparation, an estimation of the time to the target trough (5 IU FIX/dL) was made. Statistical analysis was performed by means of the Student-Fisher t-test.ResultsFifteen patients were included, nine being treated with rFIX-FP (mean age, 33 years; weight 60 kg), and six with rFIX-Fc (49 years, 86 kg). Mean DS was 3222 UI (SD, 1716) every 11,9 days (SD, 4,4) for rFIX-FP patients; and 4333 UI (SD, 606) every 14,0 days (SD, 0,0) for rFIX-Fc patients. The individual tailored DI, for a 0,05 UI/dL trough target was: applying OC; 13,6 days (SD, 5,1), -1,8 days (SD, 5,9) vs DS, representing 240 IU/day (SD, 136,1) for rFIX-FP (p=0,40), and 8,6 days (SD, 1,2), +5,4 days (SD, 1,2) vs DS, representing 508 IU/day (SD, 65,6), for rFIX-Fc, (p&lt;0,001). Applying WAPPS-HEMO; it was 15,0 days (SD, 5,1), -3,1 days (SD, 5,3) vs DS, 217 IU/day (SD, 114,7), for rFIX-FP (p=0,12), and 10,2 days (SD, 2,5), +3,8 days (SD, 2,5) vs DS, 449,7 UI/day (SD, 129,1), for rFIX-Fc, (p=0,012).Conclusion and RelevanceEfficiency of rFIX-EHL treatment following a pharmacokinetic-based tailored prophylaxis-dosing schedule versus DS in HB patients, is significantly better. Depending on the commercial preparation, rFIX-FP or rFIX-Fc. Daily-adjusted dose, for a 5 IU FIX/dL trough target, ranges between 217-240 IU/day for rFIX-FP, or 449-508 IU/day for rFIXFc, according to the two pharmacokinetic approaches (OC and population based).References and/or AcknowledgementsConflict of InterestNo conflict of interest.</description><identifier>ISSN: 2047-9956</identifier><identifier>EISSN: 2047-9964</identifier><identifier>DOI: 10.1136/ejhpharm-2023-eahp.100</identifier><language>eng</language><publisher>London: British Medical Journal Publishing Group</publisher><subject>Conflicts of interest ; Disease prevention ; Hemophilia ; Pharmacokinetics ; Section 4: Clinical pharmacy services</subject><ispartof>European journal of hospital pharmacy. Science and practice, 2023-03, Vol.30 (Suppl 1), p.A47-A48</ispartof><rights>European Association of Hospital Pharmacists 2023. No commercial re-use. See rights and permissions. Published by BMJ.</rights><rights>2023 European Association of Hospital Pharmacists 2023. No commercial re-use. See rights and permissions. Published by BMJ.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>314,777,781,27905,27906</link.rule.ids></links><search><creatorcontrib>Juárez-Giménez, JC</creatorcontrib><creatorcontrib>Benítez-Hidalgo, O</creatorcontrib><creatorcontrib>Romero-Garrido, JA</creatorcontrib><creatorcontrib>Mateos-Salillas, C</creatorcontrib><creatorcontrib>González-Piñeiro, S</creatorcontrib><creatorcontrib>Montoro-Ronsano, JB</creatorcontrib><title>4CPS-072 Real-world experience in haemophilia B patients after switching to fix extended half-life using pharmacokinetic population software and monocompartmental model</title><title>European journal of hospital pharmacy. Science and practice</title><addtitle>Eur J Hosp Pharm</addtitle><description>Background and ImportanceNew strategies have been developed for the prophylactic treatment of patients with haemophilia B (HB) such as extended half-life recombinant factor IX concentrates (rFIX EHL). These products have shown favourable pharmacokinetic properties, attaining a half-life 3- to 5-fold longer in rFIX EHL compared to standard FIX concentratesAim and ObjectivesEfficiency of a pharmacokinetic-based tailored prophylaxis-dosing schedule versus standard dosing (DS) is compared, in HB, treated with two rFIX-EHL.Material and MethodsObservational, analytical, prospective, multicentre study, involving HB patients, being treated with rFIX-EHL linked to albumin (rFIX-FP) or to fragment crystallisable (rFIX-Fc). Demographic and clinical data, and DS and dosing interval (DI) and actual FIX trough levels were recorded. Pharmacokinetic characterisation was performed following both a population (WAPPS-HEMO) and a linear one-compartment (OC) approach. For each approach and rFIX preparation, an estimation of the time to the target trough (5 IU FIX/dL) was made. Statistical analysis was performed by means of the Student-Fisher t-test.ResultsFifteen patients were included, nine being treated with rFIX-FP (mean age, 33 years; weight 60 kg), and six with rFIX-Fc (49 years, 86 kg). Mean DS was 3222 UI (SD, 1716) every 11,9 days (SD, 4,4) for rFIX-FP patients; and 4333 UI (SD, 606) every 14,0 days (SD, 0,0) for rFIX-Fc patients. The individual tailored DI, for a 0,05 UI/dL trough target was: applying OC; 13,6 days (SD, 5,1), -1,8 days (SD, 5,9) vs DS, representing 240 IU/day (SD, 136,1) for rFIX-FP (p=0,40), and 8,6 days (SD, 1,2), +5,4 days (SD, 1,2) vs DS, representing 508 IU/day (SD, 65,6), for rFIX-Fc, (p&lt;0,001). Applying WAPPS-HEMO; it was 15,0 days (SD, 5,1), -3,1 days (SD, 5,3) vs DS, 217 IU/day (SD, 114,7), for rFIX-FP (p=0,12), and 10,2 days (SD, 2,5), +3,8 days (SD, 2,5) vs DS, 449,7 UI/day (SD, 129,1), for rFIX-Fc, (p=0,012).Conclusion and RelevanceEfficiency of rFIX-EHL treatment following a pharmacokinetic-based tailored prophylaxis-dosing schedule versus DS in HB patients, is significantly better. Depending on the commercial preparation, rFIX-FP or rFIX-Fc. Daily-adjusted dose, for a 5 IU FIX/dL trough target, ranges between 217-240 IU/day for rFIX-FP, or 449-508 IU/day for rFIXFc, according to the two pharmacokinetic approaches (OC and population based).References and/or AcknowledgementsConflict of InterestNo conflict of interest.</description><subject>Conflicts of interest</subject><subject>Disease prevention</subject><subject>Hemophilia</subject><subject>Pharmacokinetics</subject><subject>Section 4: Clinical pharmacy services</subject><issn>2047-9956</issn><issn>2047-9964</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2023</creationdate><recordtype>article</recordtype><recordid>eNpFUctOwzAQjBBIIOAXkCXOBr_SJEeoeEmVQDzOkeOsiYtjm8RVe-TCz_BZfAku5XHa1c7Mzmony44oOaGUT05h3oVODj1mhHEMsgsnlJCtbI8RUeCqmojtvz6f7GaH42gaknNeVoJXe9mHmN49YFKwz7f3e5AWL_1gWwSrAIMBpwAZhzoJvQ-dsUaicxRkTEgckdQRBjQuTVSdcc8oeqTNKmkjuBbaJLMaW6MBLcY1_n2oVP7FOIhGoeDDwqZl3qHR67iUAyDpWtR755Xvgxxin4ykTZMW7EG2o6Ud4fCn7mdPlxeP02s8u726mZ7NcENpRTAXwAlpWFNQ0WjNciCKaaBlTrjSjRCKljoXrC1bIC1QwXghVZEXDRWVpBO-nx1v9obBvy5gjPXcLwaXLGtWlOmjQrAysdiG1fTzfwIl9TqW-jeWeh1LvY4lQYR_AYkhiKQ</recordid><startdate>20230323</startdate><enddate>20230323</enddate><creator>Juárez-Giménez, JC</creator><creator>Benítez-Hidalgo, O</creator><creator>Romero-Garrido, JA</creator><creator>Mateos-Salillas, C</creator><creator>González-Piñeiro, S</creator><creator>Montoro-Ronsano, JB</creator><general>British Medical Journal Publishing Group</general><general>BMJ Publishing Group LTD</general><scope>K9.</scope></search><sort><creationdate>20230323</creationdate><title>4CPS-072 Real-world experience in haemophilia B patients after switching to fix extended half-life using pharmacokinetic population software and monocompartmental model</title><author>Juárez-Giménez, JC ; Benítez-Hidalgo, O ; Romero-Garrido, JA ; Mateos-Salillas, C ; González-Piñeiro, S ; Montoro-Ronsano, JB</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-b1190-34e300b2b714bff25e0c2fe18503cfb44c18f542d8de0de14237ac757b149a163</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2023</creationdate><topic>Conflicts of interest</topic><topic>Disease prevention</topic><topic>Hemophilia</topic><topic>Pharmacokinetics</topic><topic>Section 4: Clinical pharmacy services</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Juárez-Giménez, JC</creatorcontrib><creatorcontrib>Benítez-Hidalgo, O</creatorcontrib><creatorcontrib>Romero-Garrido, JA</creatorcontrib><creatorcontrib>Mateos-Salillas, C</creatorcontrib><creatorcontrib>González-Piñeiro, S</creatorcontrib><creatorcontrib>Montoro-Ronsano, JB</creatorcontrib><collection>ProQuest Health &amp; Medical Complete (Alumni)</collection><jtitle>European journal of hospital pharmacy. Science and practice</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Juárez-Giménez, JC</au><au>Benítez-Hidalgo, O</au><au>Romero-Garrido, JA</au><au>Mateos-Salillas, C</au><au>González-Piñeiro, S</au><au>Montoro-Ronsano, JB</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>4CPS-072 Real-world experience in haemophilia B patients after switching to fix extended half-life using pharmacokinetic population software and monocompartmental model</atitle><jtitle>European journal of hospital pharmacy. Science and practice</jtitle><stitle>Eur J Hosp Pharm</stitle><date>2023-03-23</date><risdate>2023</risdate><volume>30</volume><issue>Suppl 1</issue><spage>A47</spage><epage>A48</epage><pages>A47-A48</pages><issn>2047-9956</issn><eissn>2047-9964</eissn><abstract>Background and ImportanceNew strategies have been developed for the prophylactic treatment of patients with haemophilia B (HB) such as extended half-life recombinant factor IX concentrates (rFIX EHL). These products have shown favourable pharmacokinetic properties, attaining a half-life 3- to 5-fold longer in rFIX EHL compared to standard FIX concentratesAim and ObjectivesEfficiency of a pharmacokinetic-based tailored prophylaxis-dosing schedule versus standard dosing (DS) is compared, in HB, treated with two rFIX-EHL.Material and MethodsObservational, analytical, prospective, multicentre study, involving HB patients, being treated with rFIX-EHL linked to albumin (rFIX-FP) or to fragment crystallisable (rFIX-Fc). Demographic and clinical data, and DS and dosing interval (DI) and actual FIX trough levels were recorded. Pharmacokinetic characterisation was performed following both a population (WAPPS-HEMO) and a linear one-compartment (OC) approach. For each approach and rFIX preparation, an estimation of the time to the target trough (5 IU FIX/dL) was made. Statistical analysis was performed by means of the Student-Fisher t-test.ResultsFifteen patients were included, nine being treated with rFIX-FP (mean age, 33 years; weight 60 kg), and six with rFIX-Fc (49 years, 86 kg). Mean DS was 3222 UI (SD, 1716) every 11,9 days (SD, 4,4) for rFIX-FP patients; and 4333 UI (SD, 606) every 14,0 days (SD, 0,0) for rFIX-Fc patients. The individual tailored DI, for a 0,05 UI/dL trough target was: applying OC; 13,6 days (SD, 5,1), -1,8 days (SD, 5,9) vs DS, representing 240 IU/day (SD, 136,1) for rFIX-FP (p=0,40), and 8,6 days (SD, 1,2), +5,4 days (SD, 1,2) vs DS, representing 508 IU/day (SD, 65,6), for rFIX-Fc, (p&lt;0,001). Applying WAPPS-HEMO; it was 15,0 days (SD, 5,1), -3,1 days (SD, 5,3) vs DS, 217 IU/day (SD, 114,7), for rFIX-FP (p=0,12), and 10,2 days (SD, 2,5), +3,8 days (SD, 2,5) vs DS, 449,7 UI/day (SD, 129,1), for rFIX-Fc, (p=0,012).Conclusion and RelevanceEfficiency of rFIX-EHL treatment following a pharmacokinetic-based tailored prophylaxis-dosing schedule versus DS in HB patients, is significantly better. Depending on the commercial preparation, rFIX-FP or rFIX-Fc. Daily-adjusted dose, for a 5 IU FIX/dL trough target, ranges between 217-240 IU/day for rFIX-FP, or 449-508 IU/day for rFIXFc, according to the two pharmacokinetic approaches (OC and population based).References and/or AcknowledgementsConflict of InterestNo conflict of interest.</abstract><cop>London</cop><pub>British Medical Journal Publishing Group</pub><doi>10.1136/ejhpharm-2023-eahp.100</doi><oa>free_for_read</oa></addata></record>
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subjects Conflicts of interest
Disease prevention
Hemophilia
Pharmacokinetics
Section 4: Clinical pharmacy services
title 4CPS-072 Real-world experience in haemophilia B patients after switching to fix extended half-life using pharmacokinetic population software and monocompartmental model
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