Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS)
Abstract Background Limited real-world data from routine clinical care are available on the safety and effectiveness of treatment with taliglucerase alfa in patients with Gaucher disease (GD). Methods Taliglucerase Alfa Surveillance (TALIAS), a multinational prospective Drug Registry of patients wit...
| Main Authors: | , , , , , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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BMC
2022-04-01
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| Series: | Orphanet Journal of Rare Diseases |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s13023-022-02289-7 |
| _version_ | 1828230424840634368 |
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| author | Lina Titievsky Tilman Schuster Ronnie Wang Muhammad Younus Andrew Palladino Kabir Quazi Michael P. Wajnrajch Betina Hernandez Pamela S. Becker Neal J. Weinreb Christina Chambers Roy Mansfield Louise Taylor Li-Jung Tseng Paige Kaplan |
| author_facet | Lina Titievsky Tilman Schuster Ronnie Wang Muhammad Younus Andrew Palladino Kabir Quazi Michael P. Wajnrajch Betina Hernandez Pamela S. Becker Neal J. Weinreb Christina Chambers Roy Mansfield Louise Taylor Li-Jung Tseng Paige Kaplan |
| author_sort | Lina Titievsky |
| collection | DOAJ |
| description | Abstract Background Limited real-world data from routine clinical care are available on the safety and effectiveness of treatment with taliglucerase alfa in patients with Gaucher disease (GD). Methods Taliglucerase Alfa Surveillance (TALIAS), a multinational prospective Drug Registry of patients with GD, was established to evaluate the long-term safety (primary objective) and effectiveness (secondary objective) of taliglucerase alfa. We present an interim analysis of the data from the Drug Registry collected over the 5-year period from September 2013 to January 2019. Results A total of 106 patients with GD (15.1% children aged < 18 years; 53.8% females) treated with taliglucerase alfa have been enrolled in the Drug Registry, as of January 7, 2019. The median duration of follow-up was 795 days with quartiles (Q1, Q3) of 567 and 994 days. Fifty-three patients (50.0%) were from Israel, 28 (26.4%) were from the United States, and 25 (23.6%) were from Albania. At the time of enrollment, most patients (87.7%) had received prior enzyme replacement therapy (ERT). Thirty-nine of the 106 patients had treatment-emergent adverse events (AEs). Twelve of the 106 patients experienced serious AEs; two patients experienced four treatment-related serious AEs. Four patients died, although none of the deaths was considered to be related to taliglucerase alfa treatment by the treating physicians. Nine patients discontinued from the study, including the four who died. At baseline, patients with prior ERT had a higher mean hemoglobin concentration and platelet counts than treatment-naïve patients, likely reflecting the therapeutic effects of prior treatments. During follow-up, the hemoglobin concentration and platelet counts increased in the treatment-naïve patients and remained relatively constant or increased slightly in patients with prior ERT. Spleen and liver volumes decreased in treatment-naïve patients. Conclusions The interim data showed no new or emergent safety signals. The overall interim data are consistent with the clinical program experience and known safety and effectiveness profile of taliglucerase alfa. |
| first_indexed | 2024-04-12T18:48:16Z |
| format | Article |
| id | doaj.art-a2312ba99d1246d0b5b5524f11be215f |
| institution | Directory Open Access Journal |
| issn | 1750-1172 |
| language | English |
| last_indexed | 2024-04-12T18:48:16Z |
| publishDate | 2022-04-01 |
| publisher | BMC |
| record_format | Article |
| series | Orphanet Journal of Rare Diseases |
| spelling | doaj.art-a2312ba99d1246d0b5b5524f11be215f2022-12-22T03:20:33ZengBMCOrphanet Journal of Rare Diseases1750-11722022-04-0117111110.1186/s13023-022-02289-7Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS)Lina Titievsky0Tilman Schuster1Ronnie Wang2Muhammad Younus3Andrew Palladino4Kabir Quazi5Michael P. Wajnrajch6Betina Hernandez7Pamela S. Becker8Neal J. Weinreb9Christina Chambers10Roy Mansfield11Louise Taylor12Li-Jung Tseng13Paige Kaplan14Pfizer, Inc.Pfizer, Inc.Pfizer, Inc.Pfizer IncPfizer, Inc.Pfizer, Inc.Pfizer, Inc.Pfizer, Inc.University of California, IrvineUniversity of Miami Miller School of MedicineUniversity of California, San DiegoPfizer, Inc.Pfizer, Inc.Pfizer, Inc.The Children’s Hospital of PhiladelphiaAbstract Background Limited real-world data from routine clinical care are available on the safety and effectiveness of treatment with taliglucerase alfa in patients with Gaucher disease (GD). Methods Taliglucerase Alfa Surveillance (TALIAS), a multinational prospective Drug Registry of patients with GD, was established to evaluate the long-term safety (primary objective) and effectiveness (secondary objective) of taliglucerase alfa. We present an interim analysis of the data from the Drug Registry collected over the 5-year period from September 2013 to January 2019. Results A total of 106 patients with GD (15.1% children aged < 18 years; 53.8% females) treated with taliglucerase alfa have been enrolled in the Drug Registry, as of January 7, 2019. The median duration of follow-up was 795 days with quartiles (Q1, Q3) of 567 and 994 days. Fifty-three patients (50.0%) were from Israel, 28 (26.4%) were from the United States, and 25 (23.6%) were from Albania. At the time of enrollment, most patients (87.7%) had received prior enzyme replacement therapy (ERT). Thirty-nine of the 106 patients had treatment-emergent adverse events (AEs). Twelve of the 106 patients experienced serious AEs; two patients experienced four treatment-related serious AEs. Four patients died, although none of the deaths was considered to be related to taliglucerase alfa treatment by the treating physicians. Nine patients discontinued from the study, including the four who died. At baseline, patients with prior ERT had a higher mean hemoglobin concentration and platelet counts than treatment-naïve patients, likely reflecting the therapeutic effects of prior treatments. During follow-up, the hemoglobin concentration and platelet counts increased in the treatment-naïve patients and remained relatively constant or increased slightly in patients with prior ERT. Spleen and liver volumes decreased in treatment-naïve patients. Conclusions The interim data showed no new or emergent safety signals. The overall interim data are consistent with the clinical program experience and known safety and effectiveness profile of taliglucerase alfa.https://doi.org/10.1186/s13023-022-02289-7Taliglucerase alfaGaucher diseaseEffectivenessNon-interventional studySafety |
| spellingShingle | Lina Titievsky Tilman Schuster Ronnie Wang Muhammad Younus Andrew Palladino Kabir Quazi Michael P. Wajnrajch Betina Hernandez Pamela S. Becker Neal J. Weinreb Christina Chambers Roy Mansfield Louise Taylor Li-Jung Tseng Paige Kaplan Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS) Orphanet Journal of Rare Diseases Taliglucerase alfa Gaucher disease Effectiveness Non-interventional study Safety |
| title | Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS) |
| title_full | Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS) |
| title_fullStr | Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS) |
| title_full_unstemmed | Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS) |
| title_short | Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS) |
| title_sort | safety and effectiveness of taliglucerase alfa in patients with gaucher disease an interim analysis of real world data from a multinational drug registry talias |
| topic | Taliglucerase alfa Gaucher disease Effectiveness Non-interventional study Safety |
| url | https://doi.org/10.1186/s13023-022-02289-7 |
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