Ambrose Emmanuela E, Kidenya Benson R, Charles Mwesige, Ndunguru Joyce, Jonathan Agnes, Makani Julie, Minja Irene K, Ruggajo Paschal, Balandya Emmanuel
Department of Paediatrics and Child Health, Catholic University of Health and Allied Sciences, Mwanza, Tanzania.
Department of Biochemistry and Molecular Biology, Catholic University of Health and Allied Sciences, Mwanza, Tanzania.
J Blood Med. 2023 Jan 21;14:37-47. doi: 10.2147/JBM.S380901. eCollection 2023.
To assess clinical and haematological outcomes of Hydroxyurea accessed via various access means and uncover the barriers to its utilization in children with Sickle cell anaemia (SCA), North-western Tanzania.
A retrospective study was conducted between October 2020 and April 2021 at Bugando Medical Centre (BMC) through review of medical files to compare the clinical and haematological outcomes among children with SCA at baseline and followed up retrospectively for at least one year of hydroxyurea utilization, accessed via cash, insurance and projects. Subsequently, a cross-sectional survey was conducted among parents and caregivers to ascertain the barriers to access of hydroxyurea via the various means. The p-values <0.05 were considered statistically significant.
We identified 87 children with SCA who were on hydroxyurea for at least one year. The median age at baseline (before hydroxyurea) was 99 [78-151] months, and 52/87 (59.8%) were male. Compared to baseline, there was a significant reduction in proportion of patients reporting vaso-occlusive crisis, admissions and blood transfusions, a significant increase in Haemoglobin and mean corpuscular volume, conversely a significant reduction in absolute neutrophil and reticulocytes to both insurance and project participants. There was no significant change in most of these parameters among patients who accessed hydroxyurea via cash. Further, a total of 24/87 (27.6%) participants reported different barriers to access of hydroxyurea, where 10/24 (41.7%) reported hydroxyurea to be very expensive, 10/24 (41.7%) reported insurance challenges, and 4/21 (16.6%) reported unavailability of the drug.
The paediatric patients utilizing hydroxyurea accessed via insurance and projects, but not cash, experienced significant improvement in the clinical and haematological outcomes. Several barriers for access to hydroxyurea were observed which appeared to impact these outcomes. These findings call for concerted efforts to improve the sustainable access to hydroxyurea among all patients with SCA.
评估通过不同途径获取羟基脲的临床和血液学结果，并找出坦桑尼亚西北部镰状细胞贫血（SCA）患儿使用羟基脲的障碍。
2020年10月至2021年4月在布甘多医疗中心（BMC）进行了一项回顾性研究，通过查阅病历，比较SCA患儿在基线时以及回顾性随访至少一年使用羟基脲后的临床和血液学结果，羟基脲通过现金、保险和项目等途径获取。随后，对家长和照顾者进行了横断面调查，以确定通过各种途径获取羟基脲的障碍。p值<0.05被认为具有统计学意义。
我们确定了87名使用羟基脲至少一年的SCA患儿。基线（使用羟基脲前）的中位年龄为99[78 - 151]个月，52/87（59.8%）为男性。与基线相比，报告血管闭塞性危机、住院和输血的患者比例显著降低，血红蛋白和平均红细胞体积显著增加，相反，保险和项目参与者的绝对中性粒细胞和网织红细胞显著减少。通过现金获取羟基脲的患者中，这些参数大多没有显著变化。此外，共有24/87（27.6%）的参与者报告了获取羟基脲的不同障碍，其中10/24（41.7%）报告羟基脲非常昂贵，10/24（41.7%）报告了保险方面的问题，4/21（16.6%）报告药物无法获取。
通过保险和项目而非现金获取羟基脲的儿科患者，其临床和血液学结果有显著改善。观察到获取羟基脲存在若干障碍，这些障碍似乎影响了这些结果。这些发现呼吁共同努力，以改善所有SCA患者可持续获取羟基脲的情况。
