Low-dose hydroxycarbamide therapy may offer similar benefit as maximum tolerated dose for children and young adults with sickle cell disease in low-middle-income settings
| Autor: | Tushar Idhate, Lewis L. Hsu, Ifeoma P Ijei, Atoyebi Wale, Yewen Qin, Livingstone Gayus Dogara, Juliana Olufunke Lawson, Abdul Aziz Hassan, Kofi A. Anie, Baba Inusa |
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| Rok vydání: | 2018 |
| Předmět: |
Adult
Pediatrics medicine.medical_specialty Maximum Tolerated Dose Anemia Sickle Cell Disease General Biochemistry Genetics and Molecular Biology Hydroxycarbamide Young Adult 03 medical and health sciences 0302 clinical medicine Quality of life Antisickling Agents medicine Humans Hydroxyurea General Pharmacology Toxicology and Pharmaceutics Young adult Child Developing Countries Poverty Response rate (survey) Dose-Response Relationship Drug General Immunology and Microbiology business.industry General Medicine Clinical trial Regimen 030220 oncology & carcinogenesis Maximum tolerated dose business 030215 immunology medicine.drug |
| Zdroj: | F1000Research. 7:1407 |
| ISSN: | 2046-1402 |
| DOI: | 10.12688/f1000research.14589.1 |
| Popis: | The multiple clinical benefits of hydroxycarbamide in sickle cell disease are supported by a large body of evidence. The maximum tolerated dose (MTD) is the regimen recommended by guidelines from a panel of National Heart, Lung, and Blood Institute (NHLBI) experts, but other dosage regimens have been used in babies (BABY-HUG) 9 to 18 months old (20 mg/kg per day) and developing countries such as India (10 mg/kg per day); however, there has been no direct comparison of the efficacy, effectiveness, or cost-effectiveness of these different regimens. The purpose of this review was to investigate the current situation with various hydroxycarbamide regimens with particular relevance to low-middle-income countries. In regard to methodology, a literature review was undertaken by using multiple databases in PubMed and Google and the search terms included sickle cell disease, hydroxyurea, hydroxycarbamide, sickle cell anaemia, low-middle-income countries, Sub-Saharan Africa, and India. Although MTD regimens have been widely used in research, especially within North America, clinical trials elsewhere tend to use fixed-dose regimens. In a survey of haematologists across Europe and Africa, 60% (75% response rate) did not use the MTD regimen for hydroxycarbamide treatment of sickle cell disease. The recommendations are (1) for practical purposes to commence using fixed-dose hydroxycarbamide in line with BABY-HUG recommendations and then (2) to consider or propose a trial comparing MTD escalation with various fixed doses and to include as end points health-related quality of life, haemoglobin F levels, adherence, and cost-effectiveness. |
| Databáze: | OpenAIRE |
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