PURPOSE: To study the long-term evolution of the bone marrow burden (BMB) score at MRI in patients with Gaucher disease (GD) under enzyme replacement therapy (ERT). MATERIAL AND METHODS: Forty patients treated for GD were retrospectively studied in a referral centre. BMB scores were assessed on spine and femur MR examinations performed between January 2003 and June 2014. The long-term evolution of the BMB scores was analyzed using a linear mixed model. RESULTS: A total of 121 MRI examinations were performed during the study period with a mean follow-up of 7.1 years ± 5.6, an average rate of 3.1 MR examinations ± 1.7 per patient and an interval of 2.3 years ± 1.1 between examinations. Patients had received ERT during 12 years on average ± 6.7. The trend of BMB scores with time decreased significantly by 15% (P = 0.008) during the total study period and 39% (P = 0.01) during the first 5 years of treatment. No changes in BMB scores were observed after five years of treatment. CONCLUSION: In Gaucher patients, the trend of MRI BMB scores with time decreased significantly under ERT the first 5 years of treatment before a long-term stabilization. KEY POINTS: • Bone marrow infiltration of Gaucher patients responds to enzyme replacement therapy • MRI BMB score decreases mainly during the first five years of treatment • MRI BMB score tends to stabilize after five years of treatment • MR examinations could be limited after five years of treatment.
PURPOSE: To study the long-term evolution of the bone marrow burden (BMB) score at MRI in patients with Gaucher disease (GD) under enzyme replacement therapy (ERT). MATERIAL AND METHODS: Forty patients treated for GD were retrospectively studied in a referral centre. BMB scores were assessed on spine and femur MR examinations performed between January 2003 and June 2014. The long-term evolution of the BMB scores was analyzed using a linear mixed model. RESULTS: A total of 121 MRI examinations were performed during the study period with a mean follow-up of 7.1 years ± 5.6, an average rate of 3.1 MR examinations ± 1.7 per patient and an interval of 2.3 years ± 1.1 between examinations. Patients had received ERT during 12 years on average ± 6.7. The trend of BMB scores with time decreased significantly by 15% (P = 0.008) during the total study period and 39% (P = 0.01) during the first 5 years of treatment. No changes in BMB scores were observed after five years of treatment. CONCLUSION: In Gaucher patients, the trend of MRI BMB scores with time decreased significantly under ERT the first 5 years of treatment before a long-term stabilization. KEY POINTS: • Bone marrow infiltration of Gaucher patients responds to enzyme replacement therapy • MRI BMB score decreases mainly during the first five years of treatment • MRI BMB score tends to stabilize after five years of treatment • MR examinations could be limited after five years of treatment.
Authors: Neal J Weinreb; Hans C Andersson; Maryam Banikazemi; John Barranger; Ernest Beutler; Joel Charrow; Gregory A Grabowski; Carla E M Hollak; Paige Kaplan; Henry Mankin; Pramod K Mistry; Barry E Rosenbloom; Stephan Vom Dahl; Ari Zimran Journal: Arch Intern Med Date: 2008-02-11
Authors: N W Barton; R O Brady; J M Dambrosia; A M Di Bisceglie; S H Doppelt; S C Hill; H J Mankin; G J Murray; R I Parker; C E Argoff Journal: N Engl J Med Date: 1991-05-23 Impact factor: 91.245
Authors: L W Poll; J A Koch; S vom Dahl; R Willers; A Scherer; D Boerner; C Niederau; D Häussinger; U Mödder Journal: Skeletal Radiol Date: 2001-09 Impact factor: 2.199
Authors: L A Johnson; B E Hoppel; E L Gerard; S P Miller; S H Doppelt; G C Zirzow; D I Rosenthal; J M Dambrosia; S C Hill; R O Brady Journal: Radiology Date: 1992-02 Impact factor: 11.105
Authors: Andrew J Degnan; Victor M Ho-Fung; Rebecca C Ahrens-Nicklas; Christian A Barrera; Suraj D Serai; Dah-Jyuu Wang; Can Ficicioglu Journal: Insights Imaging Date: 2019-07-10