The controversial and changing role of haematopoietic cell transplantation for lysosomal storage disorders: an update.

Haematopoietic cell transplantation (HCT) can benefit some selected subsets of patients with lysosomal diseases. Results had been impressive in children with MPS I-H, but poor in other disorders. Careful, multidisciplinary decision-making regarding whether to recommend HCT and how to provide optimal pre- and post-HCT care has proven essential to increase the likelihood of a good outcome.

Hematopoietic cell transplantation activity in Europe for inherited metabolic diseases: open issues and future directions.

For the past two decades, hematopoietic cell transplantation (HCT) has been used as effective therapy for selected inherited metabolic diseases (IMD). The primary goals of this therapy have been to promote long-term survival with donor-derived engraftment and to optimize quality of life. Careful, multidisciplinary decision-making regarding whether to recommend HCT and how to provide optimal peri- and post-HCT care has proven essential to increase the likelihood of a good outcome. Guidelines for HCT and monitoring have recently been provided in this journal. Here we report data on transplant activity for IMD in Europe and briefly discuss future directions. It is imperative that data collection for these procedures becomes as routine as that for patients undergoing HCT for malignancy and that follow-up is performed in a systematic manner. Large clinical trials have never been performed in this transplant field. Fortunately, accreditation procedures and improvements in information technology can now provide a firm foundation for such trials, which are urgently needed.