Department of Orthopaedics, Interdisciplinary Orthopedics, Aalborg University Hospital, Aalborg, Nordjylland, Denmark.
J Pediatr Endocrinol Metab. 2022 Jun 14;35(8):1003-1009. doi: 10.1515/jpem-2021-0775. Print 2022 Aug 26.
XLHR in children with a Rickets Severity Score ≥2 can now be treated with the new antibody drug, Burosumab, which prevents bone deformities and increases gait endurance. This study illustrates the extent of complications in the traditional orthopedic treatment of XLHR. The impact of surgery and severity of complications in this patient population has not been systematically assessed before.
The search strategy resulted in 215 studies and data were collected from 19 eligible studies and complications were categorized. Four medical charts of patients with XLHR at Aalborg University Hospital were assessed.
One complication occurred on average per surgical procedure for XLHR in the published literature. The 168 reported complications were categorized as follows: Type I (n=79): Complications with minimal intervention required and treatment goal still achieved, Type II (n=41): Complications with substantial change in treatment plan and treatment goal still achieved, Type IIIA (n=23): Complications with failure to achieve treatment goal and no new pathology or permanent sequelae, Type IIIB (n=25): Complications with failure to achieve treatment goal and/or new pathology or permanent sequelae.
In average, one complication occurred per surgery and the severity of complications were substantial. The treatment goal was not achieved in 28% of surgeries whereof half of them resulted in permanent sequalae or new pathology. Our findings support the use of Burosumab for treatment of the skeletal changes in XLHR as the reported side-effects in Burosumab treatment appear negligible compared to the impact of surgeries and related complications (Imel EA, Glorieux FH, Whyte MP, Munns CF, Ward LM, Nilsson O, et al. Burosumab versus conventional therapy in children with X-linked hypophosphataemia: a randomised, active-controlled, open-label, phase 3 trial. Lancet 2019;393:2416-27). However, orthopedic surgery might still be needed for correcting deformities restricting activities of daily living in XLHR patients.
现在,患有佝偻病严重程度评分≥2 的 XLHR 儿童可以使用新型抗体药物布罗索尤单抗进行治疗,该药物可预防骨骼畸形并提高步态耐力。本研究说明了传统矫形治疗 XLHR 中并发症的严重程度。在此患者人群中,手术的影响和并发症的严重程度尚未系统评估。
搜索策略共产生了 215 项研究,并从 19 项符合条件的研究中收集数据,并对并发症进行分类。对奥尔堡大学医院的 4 名 XLHR 患者的病历进行了评估。
在已发表的文献中,平均每例 XLHR 手术发生一种并发症。168 例报告的并发症分为以下几类:I 型(n=79):需要最少干预的并发症,且治疗目标仍可达到;II 型(n=41):需要改变治疗方案的并发症,但治疗目标仍可达到;IIIA 型(n=23):无法达到治疗目标,但无新的病理或永久性后遗症的并发症;IIIB 型(n=25):无法达到治疗目标且/或出现新的病理或永久性后遗症的并发症。
平均而言,每例手术发生一种并发症,且并发症的严重程度较大。28%的手术治疗目标未达到,其中一半导致永久性后遗症或新的病理改变。我们的研究结果支持使用布罗索尤单抗治疗 XLHR 的骨骼变化,因为布罗索尤单抗治疗的副作用似乎微不足道,与手术及相关并发症的影响相比(Imel EA、Glorieux FH、Whyte MP、Munns CF、Ward LM、Nilsson O 等人。Burosumab 与 X 连锁低磷血症儿童的常规治疗:一项随机、活性对照、开放标签、3 期试验。柳叶刀 2019;393:2416-27)。然而,XLHR 患者日常生活活动受限的畸形可能仍需要矫形手术来纠正。