Paget骨病的临床特征及唑来膦酸盐的疗效分析

Clinical features of Paget disease and curative effect of the patients treated with zoledronate

目的分析Paget骨病(PDB)的临床特点及应用唑来膦酸盐(Zol)治疗的疗效。方法回顾分析2013年1月至2018年1月北京积水潭医院内分泌科病理确诊PDB的15例患者的病历资料。按照受累骨骼不同,分为单骨型组和多骨型组;按照是否有病理性骨折,分为骨折组与非骨折组;比较分析各组间临床各项指标。于应用Zol 5 mg静脉单次输注治疗治疗2周后随诊。结果 15例患者年龄为(45. 53±12. 78)岁;男女比例为12∶3;多骨型9例,单骨型6例;病程(7. 71±6. 97)年,Zol有效率为100%,常见的不良反应为发热(100%)及低钙血症(47%)。治疗前多骨型组较单骨型组:碱性磷酸酶(ALP)、总Ⅰ型前胶原氨基端延长肽(t P1NP)及骨钙素(OC)均有明显升高(P <0. 05);治疗后多骨型组较单骨型组:t P1NP、Ⅰ型胶原羟基端肽β特殊序列(β-CTX)及OC均有明显升高(P <0. 05)。所有PDB患者治疗后较治疗前:t P1NP、Ca有明显下降(t=-3. 741、-2. 847,P=0. 007、0. 014); OC及甲状旁腺素(PTH)有明显升高(t=3. 052、3. 375,P=0. 009、0. 005)。单骨型患者治疗后较治疗前:t P1NP、Ca有明显下降(P <0. 05); PTH明显升高(P <0. 05)。多骨型患者治疗后较治疗前:t P1NP、ALP、Ca有明显下降(P <0. 05); OC及PTH有明显升高(P <0. 05)。骨折与非骨折PDB患者的比较差异均无统计学意义(P> 0. 05)。结论 PDB的发病特征为发病率低,以散发为主,患者年龄相对较轻,男性居多,多累及四肢长骨,ALP、t P1NP均有明显升高,而且多骨型高于单骨型,血钙正常或偏低,X线、全身骨扫描核素显像及骨骼病理会有相应改变,Zol治疗有效。

Objective To investigate the clinical features of Paget disease of bone(PDB)and the curative effect of the patients treated with zoledronate.Methods Fifteen patients of PDB after pathological diagnosis were involved in Department of Endocrinology of Beijing Jishuitan Hospital from January 2013 to January 2018.All 15 patients were divided into Monostotic PDB(MPDB)group and Polystotic PDB(PPDB)group according to lesion site,and divided into fracture group and non-fracture group according to pathological fracture.Clinical features of the patients in different groups were compared.Meanwhile,clinical indices of15cases following zoledronate5mg single infusion(Zol5mg SF)were analyzed after2weeks.Results The age of15patients was(45.53±12.78)years.The proportion of men and women was12/3;9patients were involved in PPDB group and6patients were involved in MPDB group.The disease course was(7.71±6.97)years.The efficacy of Zol5mg SF is100%.The most common side reactions were fever(100%)and hypocalcemia(100%).Before treatment,alkaline phosphatase(ALP),total procollagen typeⅠamino-terminal propeptide(tP1NP)and osteocalcin(OC)of PPDB group were higher than that of MPDB group(P<0.05).The tP1NP,β-CTX and OC were significantly higher than that of MPDB group(P<0.05)after treatment.The level of tP1NP and Ca of all patients with PDB after treatment were significantly lower than those before treatment(t=-3.741,-2.947,P=0.007,0.014).Conversely,the level of OC and PTH had a significantly increase than before(t=3.052,3.375,P=0.009,0.005).Compared with those before treatment,tP1NP and Ca were significantly decreased(P<0.05).PTH was significantly increased(P<0.05)in MPDB group.Compared with those before treatment:tP1NP,ALP and Ca decreased significantly(P<0.05);OC and PTH increased significantly in PPDB group(P<0.05).There was no significant difference between fracture group and non-fracture group.Conclusion The clinical features of PDB are low incidence,mostly sporadic patients,relative young patients and mostly male;limbs are most common lesion sites;ALP and tP1NP are significantly increased;Polystotic PDB is higher than Monostotic PDB;blood calcium is normal or low;whole bone scan radionuclide imaging and skeletal pathology will have corresponding changes;Zol treatment is effective.