Hydroxyurea treatment for adult sickle cell anemia patients in Kinshasa
Abstract Background: Despite a high incidence of sickle cell anemia, hydroxyurea (HU) treatment is rarely used in the DR Congo. This study aims to assess the efficacy of HU, the incidence of side effects that may limit its use in adults and to determine the dose needed for clinical improvement in pa...
| Main Authors: | , , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Wiley
2023-08-01
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| Series: | eJHaem |
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| Online Access: | https://doi.org/10.1002/jha2.735 |
| _version_ | 1827818423550214144 |
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| author | Paul Lumbala Kabuyi Gloire Mbayabo Mamy Ngole Aimé Lumaka Zola Valerie Race Gert Matthijs Chris Van Geet Prosper Lukusa Tshilobo Koenraad Devriendt Tite Minga Mikobi |
| author_facet | Paul Lumbala Kabuyi Gloire Mbayabo Mamy Ngole Aimé Lumaka Zola Valerie Race Gert Matthijs Chris Van Geet Prosper Lukusa Tshilobo Koenraad Devriendt Tite Minga Mikobi |
| author_sort | Paul Lumbala Kabuyi |
| collection | DOAJ |
| description | Abstract Background: Despite a high incidence of sickle cell anemia, hydroxyurea (HU) treatment is rarely used in the DR Congo. This study aims to assess the efficacy of HU, the incidence of side effects that may limit its use in adults and to determine the dose needed for clinical improvement in patients. Methods: In a prospective study, patients received an initial dose of 15 mg/kg/day which was increased by 5 mg/kg every 6 months, up to a maximum of 30 mg/kg/day. The response and side effects to HU were evaluated biologically and clinically every 3 months during a 2‐year period. Results: Seventy adult patients with a moderate or severe clinical phenotype initiated treatment. Only minor side effects were reported. At the end of the 2‐year treatment phase, 45 (64.3%) had dropped out, of whom 33 were without a clear reason. Clinical and biological improvement was more marked during the first year. There was a reduction in severe vaso‐occlusive crises (p < 0.001), need for transfusion (p < 0.001), and hospitalization days (p = 0.038). Fetal hemoglobin (HbF) levels increased on average 2.9 times after 12 months (p < 0.001). The increase in mean corpuscular volume was greater in the first year (p < 0.001) than in the second year (p = 0.041). The decrease in leukocytes (p < 0.001) was significant during the first year. In 70% of patients, the 20 mg/kg/day dose was needed to reach the 20% HbF threshold. Conclusion: HU is effective and well tolerated. The magnitude of the response varies from one patient to another. Improvement of clinical manifestations is achieved in most patients with a relatively low dose. Effective implementation of HU treatment will require improved adherence to treatment. |
| first_indexed | 2024-03-12T00:47:30Z |
| format | Article |
| id | doaj.art-dab20ddfd3a84212b5cada2b70d7092d |
| institution | Directory Open Access Journal |
| issn | 2688-6146 |
| language | English |
| last_indexed | 2024-03-12T00:47:30Z |
| publishDate | 2023-08-01 |
| publisher | Wiley |
| record_format | Article |
| series | eJHaem |
| spelling | doaj.art-dab20ddfd3a84212b5cada2b70d7092d2023-09-14T15:30:47ZengWileyeJHaem2688-61462023-08-014359560110.1002/jha2.735Hydroxyurea treatment for adult sickle cell anemia patients in KinshasaPaul Lumbala Kabuyi0Gloire Mbayabo1Mamy Ngole2Aimé Lumaka Zola3Valerie Race4Gert Matthijs5Chris Van Geet6Prosper Lukusa Tshilobo7Koenraad Devriendt8Tite Minga Mikobi9Department of Pediatrics University of Kinshasa KinshasaDemocratic Republic of the CongoDepartment of Pediatrics University of Kinshasa KinshasaDemocratic Republic of the CongoCenter of Human Genetics Faculty of Medicine University of Kinshasa KinshasaDemocratic Republic of the CongoCenter of Human Genetics Faculty of Medicine University of Kinshasa KinshasaDemocratic Republic of the CongoCenter for Human Genetics Katholieke Universiteit Leuven and University Hospitals Leuven LeuvenBelgiumCenter for Human Genetics Katholieke Universiteit Leuven and University Hospitals Leuven LeuvenBelgiumDepartment of Cardiovascular Sciences Center for Molecular and Vascular Biology Katholieke Universiteit Leuven LeuvenBelgiumCenter of Human Genetics Faculty of Medicine University of Kinshasa KinshasaDemocratic Republic of the CongoCenter for Human Genetics Katholieke Universiteit Leuven and University Hospitals Leuven LeuvenBelgiumCenter of Human Genetics Faculty of Medicine University of Kinshasa KinshasaDemocratic Republic of the CongoAbstract Background: Despite a high incidence of sickle cell anemia, hydroxyurea (HU) treatment is rarely used in the DR Congo. This study aims to assess the efficacy of HU, the incidence of side effects that may limit its use in adults and to determine the dose needed for clinical improvement in patients. Methods: In a prospective study, patients received an initial dose of 15 mg/kg/day which was increased by 5 mg/kg every 6 months, up to a maximum of 30 mg/kg/day. The response and side effects to HU were evaluated biologically and clinically every 3 months during a 2‐year period. Results: Seventy adult patients with a moderate or severe clinical phenotype initiated treatment. Only minor side effects were reported. At the end of the 2‐year treatment phase, 45 (64.3%) had dropped out, of whom 33 were without a clear reason. Clinical and biological improvement was more marked during the first year. There was a reduction in severe vaso‐occlusive crises (p < 0.001), need for transfusion (p < 0.001), and hospitalization days (p = 0.038). Fetal hemoglobin (HbF) levels increased on average 2.9 times after 12 months (p < 0.001). The increase in mean corpuscular volume was greater in the first year (p < 0.001) than in the second year (p = 0.041). The decrease in leukocytes (p < 0.001) was significant during the first year. In 70% of patients, the 20 mg/kg/day dose was needed to reach the 20% HbF threshold. Conclusion: HU is effective and well tolerated. The magnitude of the response varies from one patient to another. Improvement of clinical manifestations is achieved in most patients with a relatively low dose. Effective implementation of HU treatment will require improved adherence to treatment.https://doi.org/10.1002/jha2.735adult patientshydroxyureaKinshasasickle cell anemia |
| spellingShingle | Paul Lumbala Kabuyi Gloire Mbayabo Mamy Ngole Aimé Lumaka Zola Valerie Race Gert Matthijs Chris Van Geet Prosper Lukusa Tshilobo Koenraad Devriendt Tite Minga Mikobi Hydroxyurea treatment for adult sickle cell anemia patients in Kinshasa eJHaem adult patients hydroxyurea Kinshasa sickle cell anemia |
| title | Hydroxyurea treatment for adult sickle cell anemia patients in Kinshasa |
| title_full | Hydroxyurea treatment for adult sickle cell anemia patients in Kinshasa |
| title_fullStr | Hydroxyurea treatment for adult sickle cell anemia patients in Kinshasa |
| title_full_unstemmed | Hydroxyurea treatment for adult sickle cell anemia patients in Kinshasa |
| title_short | Hydroxyurea treatment for adult sickle cell anemia patients in Kinshasa |
| title_sort | hydroxyurea treatment for adult sickle cell anemia patients in kinshasa |
| topic | adult patients hydroxyurea Kinshasa sickle cell anemia |
| url | https://doi.org/10.1002/jha2.735 |
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