20 Mar Outcomes of transplantation using various hematopoietic cell sources in children with Hurler’s syndrome after myeloablative conditioning

MedicalResearch.com Interview with Dr. Boelens

Pediatric Blood and Marrow Transplantation Program,
University Medical Center Utrecht, Utrecht, Netherlands

MedicalResearch.com: What are the main findings of the study?

Dr. Boelens: For children with Hurler’s syndrome, the receipt of a hematopoietic cell transplant (HCT) early in life with the best available human leukocyte antigen (HLA)-matched donor offers the best event free survival (EFS). Also, HCT with a well matched unrelated cord blood unit is particularly attractive as the unit is readily available.

MedicalResearch.com: Were any of the findings unexpected?

Dr. Boelens: An interesting observation is that within the group of cord blood recipients almost all have full donor chimerism associated with normal enzyme levels in leukocytes. Higher enzymes are suggested to be associated with better long term outcomes (e.g. neurodevelomental outcome, skeletal outcome). These long term outcomes are currently being studied in an international long term outcome study.

MedicalResearch.com: What should clinicians and patients take away from your report?

Dr. Boelens: Cord blood is a good alternative cell source for transplantation in Hurler’s syndrome. Patients who received full matched cord blood have similar EFS rates as patients who received cells from matched sibling donors. The fact that cord blood units are readily available makes it particularly attractive.

MedicalResearch.com: What recommendations do you have for future research as a result of this study?

Dr. Boelens: Currently (as mentioned above) we are in the analysis phase of an international long-term outcome study for Hurler’s syndrome. The results may further improve the guidelines for HCT in Hurler’s syndrome. These outcomes are also of importance to compare the outcomes with novel emerging therapies, such as gene therapy, compared therapies (e.g., cell therapies + enzyme replacement therapy etc). For Hurler’s syndrome and other rare metabolic (or related diseases), good functioning networks are of utmost importance to bring the field forward. Within these networks prospective studies can be performed much easier.

Citation:

Outcomes of transplantation using various hematopoietic cell sources in children with Hurler’s syndrome after myeloablative conditioning

Jaap Jan Boelens, Mieke Aldenhoven, Duncan Purtill, Annalisa Ruggeri, Todd DeFor,  Robert Wynn, Ed Wraith, Marina Cavazzana-Calvo, Attilio Rovelli, Alain Fischer, Jakub Tolar, Vinod K. Prasad, Maria Escolar, Eliane Gluckman, Anne O’Meara, Paul J. Orchard, Paul Veys, Mary Eapen, Joanne Kurtzberg, and Vanderson Rocha

Blood blood-2012-09-455238; published ahead of print March 14, 2013,
doi:10.1182/blood-2012-09-455238

Last Updated on November 26, 2014 by Marie Benz MD FAAD