14800-DvRappard

167 Discussion, summary and future perspectives 10 wall and a small collapsed gallbladder often found in MLD patients, there is a substantial risk of missing a polyp on ultrasound, with an increased risk of evolving into carcinoma . To minimalize this risk, we therefore advice considering cholecystectomy when polyps cannot be ruled out on the ultrasound. Kim et al 9 also report a large MLD patient cohort with a high incidence of gallbladder abnormalities. Remarkably, they do not recommend to add an ultrasound to the standard clinical care of patients, but only for patients who present with abdominal pain. Additionally, they only advise cholecystectomy for patients with gallbladder abnormalities in the setting of relevant clinical symptoms, and not a prophylactic cholecystectomy in the case of asymptomatic polyps found on ultrasound. Follow-up of transplanted patients A standard, uniform treatment and follow-up protocol would be beneficial for patient care. Our current protocol contains a first assessment for transplanted patients 6months after HCT, including neurological examination with scoring of gross motor function (GMFC-MLD), brain MRI (rated by the MLD-Loes score) including MRS, measurement of ASA activity, sulfatide excretion in urine and assessment of nerve conduction velocity. These assessments are repeated a year after HCT and consequently each year until 5 years after HCT. Cognitive function is evaluated one year after HCT, depending on age of the patient through the Bayley Scales of Infant Development-II, the Wechsler Intelligence Scale for Children-III or the Wechsler Adult Intelligence Scale-III. Chimerism analysis is usually performed at day 60 after HCT and subsequently every year after HCT, at least during the first 5 years. After that time, follow-up is adapted per patient and clinical status. As previously stated, screening of the gallbladder by abdominal ultrasound should be added to the standard clinical care of MLD patients and included in the general evaluation prior to HCT; follow-up depends on the findings, but even in patients with normal ultrasound it should be repeated every 2 or 3 years. We see ovarian dysfunction after chemotherapy in a substantial number of our female HCT-treated patients. Hormonal substitution is advised to prevent or treat symptoms related to estrogen deficiency such as osteoporosis and climacteric symptoms. Bone density should be followed as well.