Page 124 - 《中国药房》2023年19期
P. 124

5 总结                                                     atrophy[J]. N Engl J Med,2017,377(18):1723-1732.
              DTP药房是利司扑兰的重要采购渠道,DTP药房药                        [ 8 ]  MERCURI  E,DARRAS  B  T,CHIRIBOGA  C A,et  al.
          师通过规范患者用药行为、定期疗效评估、生活质量评                                 Nusinersen versus sham control in later-onset spinal mu-
                                                                   scular atrophy[J]. N Engl J Med,2018,378(7):625-635.
          估、不良反应监测等方式对患者用药全过程进行监督,
                                                              [ 9 ]  POIRIER A,WEETALL M,HEINIG K,et al. Risdiplam
          及时给予患者用药指导和收集用药效果反馈,有利于改
                                                                   distributes and increases SMN protein in both the central
          善患者临床结局。强化 DTP 药房药师对利司扑兰用药
                                                                   nervous  system  and  peripheral  organs[J].  Pharmacol  Res
          管理服务意识、提升 DTP 药房药师的专业服务能力,对                              Perspect,2018,6(6):e00447.
          患者实施全程化利司扑兰药学服务,是提升 SMA 患者                          [10]  KAKAZU  J,WALKER  N  L,BABIN  K  C,et  al.  Ris‐
          临床疗效的重要方法。现阶段,可供参考分析的 SMA                                diplam for the use of spinal muscular atrophy[J]. Orthop
          患者使用利司扑兰的数据较少,更多利司扑兰临床研究                                 Rev,2021,13(2):25579.
          正在进行中。DTP药房药师需要持续关注SMA疾病治                           [11]  CHIRIBOGA C A. Pharmacotherapy for spinal muscular
          疗进展,及时更新SMA疾病治疗用药信息,为患者提供                                atrophy in babies and children:a review of approved and
          优质药学服务。                                                  experimental  therapies[J].  Paediatr  Drugs,2022,24(6):
          参考文献                                                     585-602.
                                                              [12]  OSMANOVIC  A,SCHREIBER-KATZ  O,PETRI  S.
          [ 1 ]  OGINO S,WILSON R B. Genetic testing and risk assess‐
                                                                   Nusinersen  wearing-off  in  adult  5q-spinal  muscular  atro‐
               ment for spinal muscular atrophy (SMA)[J]. Hum Genet,
                                                                   phy patients[J]. Brain Sci,2021,11(3):367.
               2002,111(6):477-500.
                                                              [13]  DARRAS B T,FARRAR M A,MERCURI E,et al. An in‐
          [ 2 ]  SIVARAMAKRISHNAN  M, MCCARTHY  K  D,
                                                                   tegrated safety analysis of infants and children with symp‐
               CAMPAGNE  S,et  al.  Binding  to  SMN2  pre-mRNA-
                                                                   tomatic  spinal  muscular  atrophy (SMA)  treated  with
               protein  complex  elicits  specificity  for  small  molecule
                                                                   nusinersen in seven clinical trials[J]. CNS Drugs,2019,33
               splicing modifiers[J]. Nat Commun,2017,8(1):1476.
                                                                  (9):919-932.
          [ 3 ]  胡骏,薛礼浚,邵蓉.美国专业药房研究及其对我国DTP
                                                              [14]  CHIRIBOGA  C  A,BRUNO  C,DUONG  T,et  al.  Ris‐
               药房的启示[J].卫生经济研究,2018(11):31-34.
                                                                   diplam in patients previously treated with other therapies
               HU J,XUE L J,SHAO R. Research on American profes‐
                                                                   for spinal muscular atrophy:an interim analysis from the
               sional  pharmacy  and  its  enlightenment  to  China’s  DTP
                                                                   JEWELFISH  Study[J].  Neurol  Ther,2023,12(2):
               pharmacy[J]. Health Econ Res,2018(11):31-34.
                                                                   543-557.
          [ 4 ]  中华医学会儿科学分会康复学组,中国康复医学会物理
                                                              [15]  GLANZMAN A M,MAZZONE E,MAIN M,et al. The
               治疗专委会. 脊髓性肌萎缩症康复管理专家共识[J]. 中                        Children’s Hospital of Philadelphia Infant Test of Neuro‐
               华儿科杂志,2022,60(9):883-887.                           muscular Disorders (CHOP INTEND):test development
               Subspecialty Group of Rehabilitation of the Society of Pe‐  and  reliability[J].  Neuromuscul  Disord,2010,20(3):
               diatrics of Chinese Medical Association,Physical Therapy   155-161.
               Committee of Chinese Association of Rehabilitation Medi‐  [16]  MAZZONE  E  S,MAYHEW A,MONTES  J,et  al.  Re‐
               cine.  Expert  consensus  on  rehabilitation  management  of   vised upper limb module for spinal muscular atrophy:de‐
               the  spinal  muscular  atrophy[J].  Chin  J  Pediatr,2022,60  velopment  of  a  new  module[J].  Muscle  Nerve,2017,55
              (9):883-887.                                        (6):869-874.
          [ 5 ]  MASSON  R, MAZURKIEWICZ-BEŁDZIŃSKA  M,       [17]  IANNACCONE  S  T,HYNAN  L  S,MORTON A,et  al.
               ROSE K,et al. Safety and efficacy of risdiplam in patients   The  PedsQL  in  pediatric  patients  with  spinal  muscular
               with type 1 spinal muscular atrophy (FIREFISH part 2):  atrophy:feasibility,reliability,and validity of the pediatric
               secondary  analyses  from  an  open-label  trial[J].  Lancet   quality of life inventory generic core scales and neuromus‐
               Neurol,2022,21(12):1110-1119.                       cular  module[J].  Neuromuscul  Disord,2009,19(12):
          [ 6 ]  MERCURI  E,DECONINCK  N,MAZZONE  E  S,et  al.     805-812.
               Safety and efficacy of once-daily risdiplam in type 2 and   [18]  DANY A,BARBE C,RAPIN A,et al. Construction of a
               non-ambulant type 3 spinal muscular atrophy (SUNFISH   quality of life questionnaire for slowly progressive neuro‐
               part  2):a  phase  3,double-blind,randomised,placebo-  muscular disease[J]. Qual Life Res,2015,24(11):2615-
               controlled trial[J]. Lancet Neurol,2022,21(1):42-52.  2623.
          [ 7 ]  FINKEL R S,MERCURI E,DARRAS B T,et al. Nusiner-            (收稿日期:2023-02-24  修回日期:2023-08-08)
               sen versus sham control in infantile-onset spinal muscular                         (编辑:刘明伟)






          · 2418 ·    China Pharmacy  2023 Vol. 34  No. 19                            中国药房  2023年第34卷第19期
   119   120   121   122   123   124   125   126   127   128   129