Literature DB >> 26411350

Spinal fusion as a viable treatment option for scoliosis management in Pompe disease: a postoperative 3-year follow-up.

Kazuki Kawakami1, Noriaki Kawakami2, Ayato Nohara1, Taichi Tsuji1, Tetsuya Ohara1.   

Abstract

PURPOSE: This study presents 3-year postoperative outcomes of posterior spinal correction and fusion of a patient diagnosed with late-onset Pompe disease (PD) for his progressive scoliosis.
METHODS: The patient was diagnosed for PD during his infancy. Enzyme replacement therapy (ERT) was initiated at the age of 13. First office visit for his spinal deformity was at the age of 15, and 40°, 34°, 6° spinal curvatures were seen in T6-L3, T1-6, and L3-S, respectively. Reduced pulmonary function, limited gait function and atrophied limb were documented. Initial brace treatment could not control curve progression; therefore, posterior spinal correction and fusion were performed at the age of 17.
RESULTS: Immediate preoperative curves of 55°, 42° and 23° were corrected to 18°, 26° and 7° in T6-L2, T1-T6 and L2-S, respectively. Spinal fusion was performed from T3 to L4. The patient exhibited an excessively low pulmonary function preoperatively with a VC, FVC, and %VC of 1.45 L, 1.36 L, and 35 %, respectively. This has been managed with only moderate reductions despite reduced pulmonary function from PD throughout the operative period and at 3 years. At the final follow-up, VC, FVC and %VC were 1.33 L, 1.12 L and 28.5 %, respectively.
CONCLUSION: Posterior spinal correction and fusion adequately controlled spinal curvatures for 3 years after surgery. Additionally, pulmonary function was managed throughout the follow-up period. Despite ERT, skeletal muscle and pulmonary function can still be severely affected by PD. Spinal correction and fusion is a useful method for the management of spinal curvature and pulmonary function in patients with PD.

Entities:  

Keywords:  GSD type II; Neuromuscular; Pompe disease; Posterior fusion; Pulmonary function; Scoliosis

Mesh:

Year:  2015        PMID: 26411350     DOI: 10.1007/s00586-015-4249-7

Source DB:  PubMed          Journal:  Eur Spine J        ISSN: 0940-6719            Impact factor:   3.134


  10 in total

1.  Body height correction in scoliosis patients for pulmonary function test.

Authors:  K Kono; T Asazuma; N Suzuki; T Ono
Journal:  J Orthop Surg (Hong Kong)       Date:  2000-06       Impact factor: 1.118

2.  The prevalence and impact of scoliosis in Pompe disease: lessons learned from the Pompe Registry.

Authors:  Mark Roberts; Priya S Kishnani; Ans T van der Ploeg; Wolfgang Müller-Felber; Luciano Merlini; Suyash Prasad; Laura E Case
Journal:  Mol Genet Metab       Date:  2011-08-16       Impact factor: 4.797

3.  Autophagy and lysosomes in Pompe disease.

Authors:  Tokiko Fukuda; Ashley Roberts; Meghan Ahearn; Kristien Zaal; Evelyn Ralston; Paul H Plotz; Nina Raben
Journal:  Autophagy       Date:  2006-10-05       Impact factor: 16.016

Review 4.  Pompe disease gene therapy.

Authors:  Barry J Byrne; Darin J Falk; Christina A Pacak; Sushrusha Nayak; Roland W Herzog; Melissa E Elder; Shelley W Collins; Thomas J Conlon; Nathalie Clement; Brian D Cleaver; Denise A Cloutier; Stacy L Porvasnik; Saleem Islam; Mai K Elmallah; Anatole Martin; Barbara K Smith; David D Fuller; Lee Ann Lawson; Cathryn S Mah
Journal:  Hum Mol Genet       Date:  2011-04-25       Impact factor: 6.150

5.  A meta-analysis of the efficacy of non-operative treatments for idiopathic scoliosis.

Authors:  D E Rowe; S M Bernstein; M F Riddick; F Adler; J B Emans; D Gardner-Bonneau
Journal:  J Bone Joint Surg Am       Date:  1997-05       Impact factor: 5.284

6.  Autophagy and mistargeting of therapeutic enzyme in skeletal muscle in Pompe disease.

Authors:  Tokiko Fukuda; Meghan Ahearn; Ashley Roberts; Robert J Mattaliano; Kristien Zaal; Evelyn Ralston; Paul H Plotz; Nina Raben
Journal:  Mol Ther       Date:  2006-09-27       Impact factor: 11.454

7.  Toward deconstructing the phenotype of late-onset Pompe disease.

Authors:  Angela Schüller; Stephan Wenninger; Nicola Strigl-Pill; Benedikt Schoser
Journal:  Am J Med Genet C Semin Med Genet       Date:  2012-01-17       Impact factor: 3.908

Review 8.  Pompe disease: literature review and case series.

Authors:  Majed Dasouki; Omar Jawdat; Osama Almadhoun; Mamatha Pasnoor; April L McVey; Ahmad Abuzinadah; Laura Herbelin; Richard J Barohn; Mazen M Dimachkie
Journal:  Neurol Clin       Date:  2014-08       Impact factor: 3.806

9.  Pompe disease diagnosis and management guideline.

Authors:  Priya S Kishnani; Robert D Steiner; Deeksha Bali; Kenneth Berger; Barry J Byrne; Laura E Case; Laura Case; John F Crowley; Steven Downs; R Rodney Howell; Richard M Kravitz; Joanne Mackey; Deborah Marsden; Anna Maria Martins; David S Millington; Marc Nicolino; Gwen O'Grady; Marc C Patterson; David M Rapoport; Alfred Slonim; Carolyn T Spencer; Cynthia J Tifft; Michael S Watson
Journal:  Genet Med       Date:  2006-05       Impact factor: 8.822

10.  Orthopedic management of patients with Pompe disease: a retrospective case series of 8 patients.

Authors:  Gerrit Haaker; Jürgen Forst; Raimund Forst; Albert Fujak
Journal:  ScientificWorldJournal       Date:  2014-01-02
  10 in total

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