The effectiveness of hematopoietic stem cell transplantation in treating pediatric sickle cell disease: Systematic review and meta-analysis
Background: Patients with sickle cell disease (SCD) have just one recognized curative therapy option: hematopoietic stem cell transplantation (HSCT), which results in a long-lasting improvement in the clinical phenotype. Here, we assessed the effectiveness of HSCT in treating children with SCD by a...
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| Format: | Article |
| Language: | English |
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Elsevier
2024-05-01
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| Series: | Saudi Pharmaceutical Journal |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S1319016424000999 |
| _version_ | 1797243030354264064 |
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| author | Najim Z. Alshahrani Mohammed R. Algethami |
| author_facet | Najim Z. Alshahrani Mohammed R. Algethami |
| author_sort | Najim Z. Alshahrani |
| collection | DOAJ |
| description | Background: Patients with sickle cell disease (SCD) have just one recognized curative therapy option: hematopoietic stem cell transplantation (HSCT), which results in a long-lasting improvement in the clinical phenotype. Here, we assessed the effectiveness of HSCT in treating children with SCD by a systematic review and meta-analysis. Methods: Up until January 2024, a comprehensive search was done using Web of Science, CINAHL, Embase, Google Scholar, Cochrane Library, PubMed/Medline, and Embase. Two reviewers worked separately to extract the data, and Newcastle-Ottawa Quality Assessment tool was used to assess the research's quality. The outcomes analyzed were Overall survival (OS), event-free survival (EFS), graft failure (GF) and mortality. Results: Nineteen papers satisfied our inclusion requirements and were assessed to be of fair quality. The pooled rate of OS was high (92%; 95% CI: 90.3%–93.5%). Similar finding was detected for EFS (85.8%; 95% CI: 83.7%–87.7%). In the other hand, pooled rates of GF and mortality were 6.9% (95% CI: 5.3%–8.9%) and 7.4% (95% CI: 5%–10.7%), respectively. A significant publication bias was detected for OS, EFS and GF outcomes. Subgroups analysis showed that study design was the major source of heterogeneity. Conclusion: Our results show that HSCT is effective and safe, with pooled survival rates above 90%. It is important to assess innovative tactics in light of the alarming GF and mortality rates. |
| first_indexed | 2024-04-24T18:48:38Z |
| format | Article |
| id | doaj.art-8ba19e27b44e48b48604514327ddc517 |
| institution | Directory Open Access Journal |
| issn | 1319-0164 |
| language | English |
| last_indexed | 2024-04-24T18:48:38Z |
| publishDate | 2024-05-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Saudi Pharmaceutical Journal |
| spelling | doaj.art-8ba19e27b44e48b48604514327ddc5172024-03-27T04:51:40ZengElsevierSaudi Pharmaceutical Journal1319-01642024-05-01325102049The effectiveness of hematopoietic stem cell transplantation in treating pediatric sickle cell disease: Systematic review and meta-analysisNajim Z. Alshahrani0Mohammed R. Algethami1Corresponding author: Department of Family and Community Medicine, Faculty of Medicine, University of Jeddah, Jeddah 21589, Saudi Arabia. Tel.: +966-544404833.; Department of Family and Community Medicine, University of Jeddah, Jeddah, Saudi ArabiaDepartment of Family and Community Medicine, University of Jeddah, Jeddah, Saudi ArabiaBackground: Patients with sickle cell disease (SCD) have just one recognized curative therapy option: hematopoietic stem cell transplantation (HSCT), which results in a long-lasting improvement in the clinical phenotype. Here, we assessed the effectiveness of HSCT in treating children with SCD by a systematic review and meta-analysis. Methods: Up until January 2024, a comprehensive search was done using Web of Science, CINAHL, Embase, Google Scholar, Cochrane Library, PubMed/Medline, and Embase. Two reviewers worked separately to extract the data, and Newcastle-Ottawa Quality Assessment tool was used to assess the research's quality. The outcomes analyzed were Overall survival (OS), event-free survival (EFS), graft failure (GF) and mortality. Results: Nineteen papers satisfied our inclusion requirements and were assessed to be of fair quality. The pooled rate of OS was high (92%; 95% CI: 90.3%–93.5%). Similar finding was detected for EFS (85.8%; 95% CI: 83.7%–87.7%). In the other hand, pooled rates of GF and mortality were 6.9% (95% CI: 5.3%–8.9%) and 7.4% (95% CI: 5%–10.7%), respectively. A significant publication bias was detected for OS, EFS and GF outcomes. Subgroups analysis showed that study design was the major source of heterogeneity. Conclusion: Our results show that HSCT is effective and safe, with pooled survival rates above 90%. It is important to assess innovative tactics in light of the alarming GF and mortality rates.http://www.sciencedirect.com/science/article/pii/S1319016424000999Sickle cell diseaseHematopoietic stem cell transplantationOverall survivalEvent-free survivalChildren |
| spellingShingle | Najim Z. Alshahrani Mohammed R. Algethami The effectiveness of hematopoietic stem cell transplantation in treating pediatric sickle cell disease: Systematic review and meta-analysis Saudi Pharmaceutical Journal Sickle cell disease Hematopoietic stem cell transplantation Overall survival Event-free survival Children |
| title | The effectiveness of hematopoietic stem cell transplantation in treating pediatric sickle cell disease: Systematic review and meta-analysis |
| title_full | The effectiveness of hematopoietic stem cell transplantation in treating pediatric sickle cell disease: Systematic review and meta-analysis |
| title_fullStr | The effectiveness of hematopoietic stem cell transplantation in treating pediatric sickle cell disease: Systematic review and meta-analysis |
| title_full_unstemmed | The effectiveness of hematopoietic stem cell transplantation in treating pediatric sickle cell disease: Systematic review and meta-analysis |
| title_short | The effectiveness of hematopoietic stem cell transplantation in treating pediatric sickle cell disease: Systematic review and meta-analysis |
| title_sort | effectiveness of hematopoietic stem cell transplantation in treating pediatric sickle cell disease systematic review and meta analysis |
| topic | Sickle cell disease Hematopoietic stem cell transplantation Overall survival Event-free survival Children |
| url | http://www.sciencedirect.com/science/article/pii/S1319016424000999 |
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