Curative Effect Observation of Burosumab for Children and Adults with X-Linked Hypophosphatemicrickets
We studied the patients diagnosed with X-linked hypophosphatemicrickets(XLH) and treated with burosumab in Peking Union Medical College Hospital from January 2021 to December 2022. In addition, we described the clinical characteristics of the patients, the changes of clinical indexes before and afte...
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| Format: | Article |
| Language: | zho |
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Editorial Office of Journal of Rare Diseases
2024-01-01
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| Series: | 罕见病研究 |
| Subjects: | |
| Online Access: | https://jrd.chard.org.cn/article/doi/10.12376/j.issn.2097-0501.2024.01.014 |
| _version_ | 1797289808043704320 |
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| author | LIU Qingyang HOU Zijing YANG Zixia LIU Xin ZHANG Bo TANG Yan |
| author_facet | LIU Qingyang HOU Zijing YANG Zixia LIU Xin ZHANG Bo TANG Yan |
| author_sort | LIU Qingyang |
| collection | DOAJ |
| description | We studied the patients diagnosed with X-linked hypophosphatemicrickets(XLH) and treated with burosumab in Peking Union Medical College Hospital from January 2021 to December 2022. In addition, we described the clinical characteristics of the patients, the changes of clinical indexes before and after burosumab treatment, and the adverse drug reactions during treatment. We also evaluated the efficacy and safety of burosumab for XLH. The results showed that three children XLH patients and one adult XLH patients received burosumab treatment. After treatment, the serum phosphorus level of all patients increased; the serum phosphorus of 3 children patients increased above the lower limit of the reference value range; the serum alkaline phosphatase(ALP) of all patients was lower than that of before treatment; the serum ALP of one adult patient was close to the normal range after 2.5 years of treatment. One child patient showed small crystals in kidney through ultrasound 48 weeks after treatment; one child and one adult showed increased serum parathyroid hormone(PTH)level before treatment and serum PTH continued increasing after treatment. Finally, it may be concluded that burosumab increased serum phosphorus levels in XLH patients, kept the level relatively stable, and reduced serum ALP levels. No serious adverse reactions occurred during treatment, in order to provide reference for the use of burosumab in patients with XLH. |
| first_indexed | 2024-03-07T19:11:22Z |
| format | Article |
| id | doaj.art-771278a12d2b41cb9dc577a8bb96b234 |
| institution | Directory Open Access Journal |
| issn | 2097-0501 |
| language | zho |
| last_indexed | 2024-03-07T19:11:22Z |
| publishDate | 2024-01-01 |
| publisher | Editorial Office of Journal of Rare Diseases |
| record_format | Article |
| series | 罕见病研究 |
| spelling | doaj.art-771278a12d2b41cb9dc577a8bb96b2342024-03-01T03:14:51ZzhoEditorial Office of Journal of Rare Diseases罕见病研究2097-05012024-01-013110811310.12376/j.issn.2097-0501.2024.01.014Curative Effect Observation of Burosumab for Children and Adults with X-Linked HypophosphatemicricketsLIU Qingyang0HOU ZijingYANG Zixia1LIU Xin2ZHANG Bo3TANG Yan4Department of Pharmacy, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, ChinaDepartment of Pharmacy, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, ChinaDepartment of Pharmacy, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, ChinaDepartment of Pharmacy, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, ChinaDepartment of Pharmacy, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, ChinaWe studied the patients diagnosed with X-linked hypophosphatemicrickets(XLH) and treated with burosumab in Peking Union Medical College Hospital from January 2021 to December 2022. In addition, we described the clinical characteristics of the patients, the changes of clinical indexes before and after burosumab treatment, and the adverse drug reactions during treatment. We also evaluated the efficacy and safety of burosumab for XLH. The results showed that three children XLH patients and one adult XLH patients received burosumab treatment. After treatment, the serum phosphorus level of all patients increased; the serum phosphorus of 3 children patients increased above the lower limit of the reference value range; the serum alkaline phosphatase(ALP) of all patients was lower than that of before treatment; the serum ALP of one adult patient was close to the normal range after 2.5 years of treatment. One child patient showed small crystals in kidney through ultrasound 48 weeks after treatment; one child and one adult showed increased serum parathyroid hormone(PTH)level before treatment and serum PTH continued increasing after treatment. Finally, it may be concluded that burosumab increased serum phosphorus levels in XLH patients, kept the level relatively stable, and reduced serum ALP levels. No serious adverse reactions occurred during treatment, in order to provide reference for the use of burosumab in patients with XLH.https://jrd.chard.org.cn/article/doi/10.12376/j.issn.2097-0501.2024.01.014x-linked hypophosphatemicricketsclinical manifestationsburosumabefficacysafety |
| spellingShingle | LIU Qingyang HOU Zijing YANG Zixia LIU Xin ZHANG Bo TANG Yan Curative Effect Observation of Burosumab for Children and Adults with X-Linked Hypophosphatemicrickets 罕见病研究 x-linked hypophosphatemicrickets clinical manifestations burosumab efficacy safety |
| title | Curative Effect Observation of Burosumab for Children and Adults with X-Linked Hypophosphatemicrickets |
| title_full | Curative Effect Observation of Burosumab for Children and Adults with X-Linked Hypophosphatemicrickets |
| title_fullStr | Curative Effect Observation of Burosumab for Children and Adults with X-Linked Hypophosphatemicrickets |
| title_full_unstemmed | Curative Effect Observation of Burosumab for Children and Adults with X-Linked Hypophosphatemicrickets |
| title_short | Curative Effect Observation of Burosumab for Children and Adults with X-Linked Hypophosphatemicrickets |
| title_sort | curative effect observation of burosumab for children and adults with x linked hypophosphatemicrickets |
| topic | x-linked hypophosphatemicrickets clinical manifestations burosumab efficacy safety |
| url | https://jrd.chard.org.cn/article/doi/10.12376/j.issn.2097-0501.2024.01.014 |
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