Evidence and gaps in clinical outcomes of novel pharmacologic therapies for sickle cell disease: A systematic literature review highlighting insights from clinical trials and real-world studies
<p>This systematic review aims to summarise the clinical outcomes of l-glutamine, crizanlizumab, and voxelotor in the treatment of sickle cell disease (SCD) based on clinical trials and real-world data and to identify any gaps in the observations. The review identified 97 studies reporting dat...
محفوظ في:
| المؤلف الرئيسي: | |
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| مؤلفون آخرون: | , , , , , , , , |
| منشور في: |
2025
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| الموضوعات: | |
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إضافة وسم
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| _version_ | 1864513549640925184 |
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| author | Mohamed Yassin (4166515) |
| author2 | Caterina Minniti (21189398) Nirmish Shah (6672725) Salam Alkindi (11601856) Fateen Ata (12217764) Mohammed Qari (21189401) Abdullah Al Zayed (21189404) Jaffer Altooq (18186850) Mona Al Rasheed (12040519) Maria Domenica Capellini (21189407) |
| author2_role | author author author author author author author author author |
| author_facet | Mohamed Yassin (4166515) Caterina Minniti (21189398) Nirmish Shah (6672725) Salam Alkindi (11601856) Fateen Ata (12217764) Mohammed Qari (21189401) Abdullah Al Zayed (21189404) Jaffer Altooq (18186850) Mona Al Rasheed (12040519) Maria Domenica Capellini (21189407) |
| author_role | author |
| dc.creator.none.fl_str_mv | Mohamed Yassin (4166515) Caterina Minniti (21189398) Nirmish Shah (6672725) Salam Alkindi (11601856) Fateen Ata (12217764) Mohammed Qari (21189401) Abdullah Al Zayed (21189404) Jaffer Altooq (18186850) Mona Al Rasheed (12040519) Maria Domenica Capellini (21189407) |
| dc.date.none.fl_str_mv | 2025-04-25T09:00:00Z |
| dc.identifier.none.fl_str_mv | 10.1016/j.blre.2025.101298 |
| dc.relation.none.fl_str_mv | https://figshare.com/articles/journal_contribution/Evidence_and_gaps_in_clinical_outcomes_of_novel_pharmacologic_therapies_for_sickle_cell_disease_A_systematic_literature_review_highlighting_insights_from_clinical_trials_and_real-world_studies/28881116 |
| dc.rights.none.fl_str_mv | CC BY 4.0 info:eu-repo/semantics/openAccess |
| dc.subject.none.fl_str_mv | Biomedical and clinical sciences Cardiovascular medicine and haematology Clinical sciences Pharmacology and pharmaceutical sciences Sickle cell diseasel-glutamine Crizanlizumab Voxelotor, real-world studies Clinical trials Vaso-occlusive crisis |
| dc.title.none.fl_str_mv | Evidence and gaps in clinical outcomes of novel pharmacologic therapies for sickle cell disease: A systematic literature review highlighting insights from clinical trials and real-world studies |
| dc.type.none.fl_str_mv | Text Journal contribution info:eu-repo/semantics/publishedVersion text contribution to journal |
| description | <p>This systematic review aims to summarise the clinical outcomes of l-glutamine, crizanlizumab, and voxelotor in the treatment of sickle cell disease (SCD) based on clinical trials and real-world data and to identify any gaps in the observations. The review identified 97 studies reporting data until 31 May 2024. A pivotal phase III study of l-glutamine showed that patients treated with l-glutamine had a 25 % reduction in pain crises and 33 % fewer hospital days compared to placebo. l-glutamine was generally well tolerated with minimal side effects. Real-world studies of l-glutamine emphasize patient adherence and obstacles to medication accessibility and approval as key concerns. In the SUSTAIN study, a 5-mg/kg dose of crizanlizumab reduced the occurrence of vaso-occlusive crises (VOCs) and hospitalizations by 45 % and 41 %, respectively. Real-world studies of crizanlizumab showed a reduction in complicated VOC events. The high discontinuation rate and results of the STAND trial led to a significant decrease in the use of crizanlizumab. The HOPE trial demonstrated a 51 % improvement in hemoglobin response and a reduction in hemolytic markers in patients treated with voxelotor. While some real-world studies have reported a decrease in VOCs and hospitalizations, the results are inconsistent and not conclusive. Further studies are needed to assess the impact of these novel therapies on end-organ-specific complications of SCD.</p><h2>Other Information</h2> <p> Published in: Blood Reviews<br> License: <a href="http://creativecommons.org/licenses/by/4.0/" target="_blank">http://creativecommons.org/licenses/by/4.0/</a><br>See article on publisher's website: <a href="https://dx.doi.org/10.1016/j.blre.2025.101298" target="_blank">https://dx.doi.org/10.1016/j.blre.2025.101298</a></p> |
| eu_rights_str_mv | openAccess |
| id | Manara2_57a198f586d7bb5034ce587c898f1bf5 |
| identifier_str_mv | 10.1016/j.blre.2025.101298 |
| network_acronym_str | Manara2 |
| network_name_str | Manara2 |
| oai_identifier_str | oai:figshare.com:article/28881116 |
| publishDate | 2025 |
| repository.mail.fl_str_mv | |
| repository.name.fl_str_mv | |
| repository_id_str | |
| rights_invalid_str_mv | CC BY 4.0 |
| spelling | Evidence and gaps in clinical outcomes of novel pharmacologic therapies for sickle cell disease: A systematic literature review highlighting insights from clinical trials and real-world studiesMohamed Yassin (4166515)Caterina Minniti (21189398)Nirmish Shah (6672725)Salam Alkindi (11601856)Fateen Ata (12217764)Mohammed Qari (21189401)Abdullah Al Zayed (21189404)Jaffer Altooq (18186850)Mona Al Rasheed (12040519)Maria Domenica Capellini (21189407)Biomedical and clinical sciencesCardiovascular medicine and haematologyClinical sciencesPharmacology and pharmaceutical sciencesSickle cell diseasel-glutamineCrizanlizumabVoxelotor, real-world studiesClinical trialsVaso-occlusive crisis<p>This systematic review aims to summarise the clinical outcomes of l-glutamine, crizanlizumab, and voxelotor in the treatment of sickle cell disease (SCD) based on clinical trials and real-world data and to identify any gaps in the observations. The review identified 97 studies reporting data until 31 May 2024. A pivotal phase III study of l-glutamine showed that patients treated with l-glutamine had a 25 % reduction in pain crises and 33 % fewer hospital days compared to placebo. l-glutamine was generally well tolerated with minimal side effects. Real-world studies of l-glutamine emphasize patient adherence and obstacles to medication accessibility and approval as key concerns. In the SUSTAIN study, a 5-mg/kg dose of crizanlizumab reduced the occurrence of vaso-occlusive crises (VOCs) and hospitalizations by 45 % and 41 %, respectively. Real-world studies of crizanlizumab showed a reduction in complicated VOC events. The high discontinuation rate and results of the STAND trial led to a significant decrease in the use of crizanlizumab. The HOPE trial demonstrated a 51 % improvement in hemoglobin response and a reduction in hemolytic markers in patients treated with voxelotor. While some real-world studies have reported a decrease in VOCs and hospitalizations, the results are inconsistent and not conclusive. Further studies are needed to assess the impact of these novel therapies on end-organ-specific complications of SCD.</p><h2>Other Information</h2> <p> Published in: Blood Reviews<br> License: <a href="http://creativecommons.org/licenses/by/4.0/" target="_blank">http://creativecommons.org/licenses/by/4.0/</a><br>See article on publisher's website: <a href="https://dx.doi.org/10.1016/j.blre.2025.101298" target="_blank">https://dx.doi.org/10.1016/j.blre.2025.101298</a></p>2025-04-25T09:00:00ZTextJournal contributioninfo:eu-repo/semantics/publishedVersiontextcontribution to journal10.1016/j.blre.2025.101298https://figshare.com/articles/journal_contribution/Evidence_and_gaps_in_clinical_outcomes_of_novel_pharmacologic_therapies_for_sickle_cell_disease_A_systematic_literature_review_highlighting_insights_from_clinical_trials_and_real-world_studies/28881116CC BY 4.0info:eu-repo/semantics/openAccessoai:figshare.com:article/288811162025-04-25T09:00:00Z |
| spellingShingle | Evidence and gaps in clinical outcomes of novel pharmacologic therapies for sickle cell disease: A systematic literature review highlighting insights from clinical trials and real-world studies Mohamed Yassin (4166515) Biomedical and clinical sciences Cardiovascular medicine and haematology Clinical sciences Pharmacology and pharmaceutical sciences Sickle cell diseasel-glutamine Crizanlizumab Voxelotor, real-world studies Clinical trials Vaso-occlusive crisis |
| status_str | publishedVersion |
| title | Evidence and gaps in clinical outcomes of novel pharmacologic therapies for sickle cell disease: A systematic literature review highlighting insights from clinical trials and real-world studies |
| title_full | Evidence and gaps in clinical outcomes of novel pharmacologic therapies for sickle cell disease: A systematic literature review highlighting insights from clinical trials and real-world studies |
| title_fullStr | Evidence and gaps in clinical outcomes of novel pharmacologic therapies for sickle cell disease: A systematic literature review highlighting insights from clinical trials and real-world studies |
| title_full_unstemmed | Evidence and gaps in clinical outcomes of novel pharmacologic therapies for sickle cell disease: A systematic literature review highlighting insights from clinical trials and real-world studies |
| title_short | Evidence and gaps in clinical outcomes of novel pharmacologic therapies for sickle cell disease: A systematic literature review highlighting insights from clinical trials and real-world studies |
| title_sort | Evidence and gaps in clinical outcomes of novel pharmacologic therapies for sickle cell disease: A systematic literature review highlighting insights from clinical trials and real-world studies |
| topic | Biomedical and clinical sciences Cardiovascular medicine and haematology Clinical sciences Pharmacology and pharmaceutical sciences Sickle cell diseasel-glutamine Crizanlizumab Voxelotor, real-world studies Clinical trials Vaso-occlusive crisis |