Five patients with adult-onset metachromatic leukodystrophy (MLD) underwent allo-SCT. Conditioning was reduced in intensity and grafts were obtained from voluntary unrelated donors. All but one graft were depleted of T-lymphocytes. Patient age at transplantation varied from 18 to 29 (median, 27) years. Two patients rejected their graft and MLD progressed. The recipient of the unmanipulated graft converted to complete donor chimerism with normalization of arylsulphatase A (ARSA) levels. Despite ARSA normalization, he deteriorated. Another patient was a mixed chimera. Following escalated doses of donor lymphocyte infusions he converted to complete donor chimerism. His levels of ARSA correlated positively with the percentage of donor cells and MLD was not progressive. The fifth patient died after 35 days from complications associated with GVHD. We conclude that results of allo-SCT in symptomatic MLD patients are poor. However, allo-SCT may stop progression of MLD in selected patients.

The currently used systemic treatments for GEP-NET patients had low scores according to the NHB-ASCO-F and none could be graded as meaningful clinical beneficial according to the ESMO-MCBS. Despite the low incidence, the heterogeneous patient population and relatively long natural course of NET, future studies on new treatment modalities should aim for high clinical benefit outcomes.

PurposePatients with a neuroendocrine tumor (NET) frequently experience physical and psychosocial complaints. Novel strategies to provide information to optimize supportive care in these patients are of interest. The aim of this study was to examine whether the use of a web-based system consisting of self-screening of problems and care needs, patient education, and self-referral to professional health care is feasible in NET patients and to evaluate their opinion on this.MethodsNewly diagnosed NET patients were randomized between standard care (n = 10) or intervention with additional access to the web-based system (n = 10) during 12 weeks. Patients completed questionnaires regarding received information, distress, quality of life (QoL), and empowerment. The intervention group completed a semi-structured interview to assess patients’ opinion on the web-based system.ResultsThe participation rate was 77% (20/26 invited patients) with no dropouts. The use of the web-based system had a negative effect on patients’ perception and satisfaction of received information (range Cohen’s d −0.88 to 0.13). Positive effects were found for distress (Cohen’s d 0.75), global QoL (subscale European Organization for Research and Treatment of Cancer (EORTC) QLQ-C30, Cohen’s d 0.46), resolving problems with social functioning and finding information (subscales EORTC QLQ-GINET 21, Cohen’s d 0.69, respectively, 1.04), and feeling informed (subscale empowerment questionnaire, Cohen’s d 0.51). The interview indicated that the web-based system was of additional value to standard care.ConclusionsUse of this web-based system is feasible. Contradictory effects on informing and supporting NET patients were found and should be subject of further research.Trial registration NCT01849523