不同剂型短效rhGH治疗矮小症患者的有效性、安全性及经济性评价

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中图分类号 R969；R956;R977.1 文献标志码A 文章编号 1001-0408（2025）09-1111-06

DOI 10.6039/j.issn.1001-0408.2025.09.16

ABSTRACTOBJECTVETocomparetheefcacy，safety，andcost-efectivenessof twodiferentformulationsofshort-acting recombinant human growth hormone（rhGH）in thetreatmentof patients with short stature.METHODSData frompatients with shortstaturetreatedwithshort-actingrhGHattheLeshanPeople’sHospitalfromAugust2O16toJune2023werecolectedPatients weredividedinto powder formulation groupandaqueous formulation groupbasedontherhGH formulationused.Thechanges in growth-relatedeficacyindicatorsandtheoccurrenceof adversedrugreactions werecomparedbetwentwo groupsafterl2months oftreatment；cost-effectivenessanalysisandsensitivityanalysiswereusedtocomparethecostperunitofefectacheved；subgroup analysis wasperformedbydividingthepatients intogrowthhormonedeficiency（GHD）subgroupandidiopathicshortstature（Is） subgroupbasedonclinical diagnosis.RESULTSAfter12 monthsoftreatment，theheightandthelevelsof insulin-likegrowth factor-landinsulin-likegrowthfactorbindingprotein-3inseruminaqueous formulation groupand powderformulationgroup were

significantly increased compared to before treatment（ P < 0.001），but there was no statistically significant difference in the changes of the above indicators between the two groups 1 （P>0 . 0 5 ）.Theanalysis results of GHD and ISS subgroups were consistent with the overall population. In the overall

population，the cost-effectiveness ratio of powder formulation group 2 5 8 2 y u a n / c m ）was significantly better than that of aqueous formulation group（6 729 yuan/cm），with a statistically significant difference（ P < 0 . 0 0 1 ），and the result was consistent in the GHD and ISSsubgroupsas wellasin thesensitivityanalysis.Noseriousadverse drug reactions ocurred in either powder formulationoraqueous formulationgroup，andtherewasnostatisticallysignificantdiferenceintheincidenceofvariousadverse reactions between two groups （ ΔP>0 . 0 5 ）.CONCLUSIONS Short-acting rhGH powder and aqueous formulations have equivalent efficacy and safety，but the powder formulation has greater economic advantages.

KEYWORDSrecombinant humangrowthhormone；formulation；eficacy；safety；cost-effectiveness；short stature；growth hormone deficiency；idiopathic short stature

我国矮小症患者发病率约为 3 % ，全国约有4100万例矮小症患者，但得到正规治疗的不足3万例[1-2]。（剩余8575字）