Guided growth may not be the best option for knee valgus deformity in adolescent patients with Morquio-A

Colmenares-Bonilla, Douglas; Vasconcelos-Martinez, M; Guerra-Jasso, José de Jesús; Ocampo-Perez, L

doi:10.1007/s12306-016-0441-0

Cited by 4 publications

(3 citation statements)

References 14 publications

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“…Treatment of articular cartilage pathology with AAV9-Galns gene therapy. KS accumulation alters articular cartilage in MPSIVA patients leading to severe osteoarthritis that, in time, will require orthopedic surgery 3,44,45 . In MPSIVA rats at 4 weeks of age, analysis of tibial cartilage sections with both Safranin O staining and LIMP2 immunostaining already demonstrated increased chondrocyte hypertrophy (about 2-fold) compared with WT rats, indicating early storage pathology (Supplementary Fig.…”

Section: Resultsmentioning

confidence: 99%

Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy

et al. 2021

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Mucopolysaccharidosis type IVA (MPSIVA) or Morquio A disease, a lysosomal storage disorder, is caused by N-acetylgalactosamine-6-sulfate sulfatase (GALNS) deficiency, resulting in keratan sulfate (KS) and chondroitin-6-sulfate accumulation. Patients develop severe skeletal dysplasia, early cartilage deterioration and life-threatening heart and tracheal complications. There is no cure and enzyme replacement therapy cannot correct skeletal abnormalities. Here, using CRISPR/Cas9 technology, we generate the first MPSIVA rat model recapitulating all skeletal and non-skeletal alterations experienced by patients. Treatment of MPSIVA rats with adeno-associated viral vector serotype 9 encoding Galns (AAV9-Galns) results in widespread transduction of bones, cartilage and peripheral tissues. This led to long-term (1 year) increase of GALNS activity and whole-body correction of KS levels, thus preventing body size reduction and severe alterations of bones, teeth, joints, trachea and heart. This study demonstrates the potential of AAV9-Galns gene therapy to correct the disabling MPSIVA pathology, providing strong rationale for future clinical translation to MPSIVA patients.

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Section: Resultsmentioning

confidence: 99%

Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy

et al. 2021

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“…All these Hispanic patients share ethnic characteristics which may be regionally oriented than other populations around the globe. Is the need to early diagnose these patients in order to avoid clinical deterioration and worst surgery outcomes (20).…”

Section: Definitive Diagnosismentioning

confidence: 99%

Diagnosis of Morquio-A patients in Mexico: How far are we from prompt diagnosis?

Colmenares-Bonilla

Esquitin-Garduño

2017

IRDR

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“…On the opposite side Colmenares-bonilla [19] recommends corrective osteotomy in treating patients with Morquio-A (MPS) because they usually had insufficient growth power especially when been diagnosed in adolescent age and this may help reduce arthritis progression in adjacent joints.…”

Section: Discussionmentioning

confidence: 99%

The Using of "Guided Growth" for Correction of Coronal Deformities around the Knee in Skeletally Immature Children (Systematic review and Metaanalysis)

Sawiris¹,

Abo-Seif²,

Aly³

2018

Med Clin Rev

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Introduction: Angular deformities of the lower limbs are common during childhood. The concept of guided growth using a tension band plate is a breakthrough in the surgical correction of angular deformities. This approach has yielded satisfactory results and has low complication rates. Patients and methods:We reviewed 210 papers with overall 433 patients and 809 deformities. Inclusion criteria included papers published from 2010 to 2017 in English literature. A Meta-analysis was done on 16 papers with seven outcomes: Change in mechanical axis deviation, Tibiofemoral angle, rate of excellent outcomes, implant breakage, implant migration, inadequate reduction and rebound phenomenon.Results: Thirty two studies were included. Fifteen of them studied treatment of angular deformities by guided growth, three of them discussed the complications of using guided growth and five studies discussed Biomechanics of using guided growth. Sixteen studies were included in our cumulative meta-analysis. Conclusion:Guided growth is effective in treating coronal deformities around the knee in skeletally immature children wither the cause was idiopathic or pathological. There is no limitation except if physeal bar is developed or the patient near maturity. Few complications are associated with guided growth techniques including rebound phenomenon, inadequate reduction, implant breakage and implant migration.

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Guided growth may not be the best option for knee valgus deformity in adolescent patients with Morquio-A

Abstract: This physeal osteotomy is a feasible and optimal option to achieve acute valgus correction on severe deformity when there is not enough remaining growth on adolescent Morquio-A patients, and may help reduce arthritis progression in adjacent joints.

Cited by 4 publications

References 14 publications

Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy

Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy

Diagnosis of Morquio-A patients in Mexico: How far are we from prompt diagnosis?

The Using of "Guided Growth" for Correction of Coronal Deformities around the Knee in Skeletally Immature Children (Systematic review and Metaanalysis)

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