Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis

Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially curative treatment for patients affected by Wiskott-Aldrich syndrome (WAS). Reported HSCT outcomes have improved over time with respect to overall survival, but some studies have identified older age and HSCT from alternative donors as risk factors predicting poorer outcome. We analyzed 197 patients undergoing transplant at European Society for Blood and Marrow Transplantation centers between 2006 and 2017 who received conditioning as recommended by the Inborn Errors Working Party (IEWP): either busulfan (n = 103) or treosulfan (n = 94) combined with fludarabine 6 thiotepa. After a median follow-up post-HSCT of 44.9 months, 176 patients were alive, resulting in a 3-year overall survival of 88.7% and chronic graft-versus-host disease (GVHD)-free survival (events include death, graft failure, and severe chronic GVHD) of 81.7%. Overall survival and chronic GVHD-free survival were not significantly affected by conditioning regimen (busulfan-vs treosulfan-based), donor type (matched sibling donor/matched family donor vs matched unrelated donor/mismatched unrelated donor vs mismatched family donor), or period of HSCT (2006-2013 vs 2014-2017). Patients aged = 5 years remains a risk factor for overall survival.Transplantation and immunomodulatio

Equine autologous platelet concentrates: A comparative study between different available systems

REASONS FOR PERFORMING STUDY: Autologous platelet concentrates (APCs) are being used increasingly in horses to enhance regeneration in tissues that have poor natural healing capabilities. Numerous APC systems, which are based on different preparation techniques and were originally developed for human patients, are now routinely used in equine cases. However, preliminary process validation and adequate in vitro biochemical characterisation of most of these systems do not exist for horses. OBJECTIVES: To compare haematological findings and growth factor concentrations of equine APCs obtained with 4 commercially available systems and a noncommercial double-centrifugation technique. STUDY DESIGN: Nonrandomised in vitro experiment. METHODS: Blood samples from 6 horses were processed to produce APCs using one equine-specific filtration-based and 4 different centrifugation-based techniques. Platelet, leucocyte, platelet-derived growth factor-BB and transforming growth factor-β1 concentrations were measured in all APCs, and their respective enrichment factors were compared. RESULTS: Mean platelet concentration increased in all systems in comparison to baseline; however, the mean enrichment factor, which ranged from 130% to 527% depending on the APC, was statistically significant in only 2 products. One method reduced total leucocyte counts to 9% of the baseline value, while the others had a mean fold increase varying from 116 to 663% of the baseline. Differential leucocyte count also differed between the products. Moreover, the various systems had significantly different mean growth factor enrichments (184-1255% for platelet-derived growth factor-BB and 93-560% for transforming growth factor-β1 ). CONCLUSIONS: Haematological and biochemical characteristics varied markedly among 5 techniques used in the field to produce APCs in horses. These discrepancies could have an impact on clinical outcomes, and further studies are needed to determine their influence on the quality of tissue regeneration. Clinicians should not rely on the manufacturers' data relating to human patients to select the most appropriate method for horses

Regenerative Medicine: Advances from Developmental to Degenerative Diseases

Chronic tissue and organ failure caused by an injury, disease, ageing or congenital defects represents some of the most complex therapeutic challenges and poses a significant financial healthcare burden. Regenerative medicine strategies aim to fulfil the unmet clinical need by restoring the normal tissue function either through stimulating the endogenous tissue repair or by using transplantation strategies to replace the missing or defective cells. Stem cells represent an essential pillar of regenerative medicine efforts as they provide a source of progenitors or differentiated cells for use in cell replacement therapies. Whilst significant leaps have been made in controlling the stem cell fates and differentiating them to cell types of interest, transitioning bespoke cellular products from an academic environment to off-the-shelf clinical treatments brings about a whole new set of challenges which encompass manufacturing, regulatory and funding issues. Notwithstanding the need to resolve such issues before cell replacement therapies can benefit global healthcare, mounting progress in the field has highlighted regenerative medicine as a realistic prospect for treating some of the previously incurable conditions