Farber's Lipogranulomatosis: Multimodal Therapy With Tocilizumab and Consolidative HSCT Improves Assessment, and Long-Term Outcome.

Farber's lipogranulomatosis (FL) is an autosomal recessive lipid storage disorder, arising as a consequence of genetic acid ceramidase deficiency. Clinically, it presents as severe arthritis, voice hoarseness, and widespread, painful subcutaneous nodules (SCN). For those without CNS involvement, haematopoietic stem cell transplant provides a viable option for the improvement of both respiratory and musculoskeletal morbidity.

Donor T-cell and myeloid chimerism is higher after cord blood transplant than with other cell sources.

Differences in multi-lineage and long-term donor chimerism after hematopoietic cell transplantation (HCT) from different cell sources are not well characterized, yet such information is crucial for understanding the distinct clinical utilities of each graft type. We analyzed donor chimerism outcomes in 472 pediatric allogeneic HCT recipients (2010-2020), including 115 unrelated cord blood (CB) grafts and 357 non-CB grafts (bone marrow or mobilized peripheral blood). Primary graft failure occurred more frequently in CB recipients (4.

Impact of shared HLA determinants between patient and losing cord blood unit on relapse after double cord blood transplantation.

During double umbilical cord blood transplant (DUCBT), the winning unit (WU) rejects the losing unit (LU) because of WU T cells directed against the LU mismatched HLA. This immune response might protect against relapse, especially when the patient and LU (PT-LU) share the same mismatch with the WU. To validate this hypothesis, a retrospective Eurocord study, conducted on 383 DUCBTs, focused on posttransplant relapse and HLA mismatches between PT-LU and the WU.

Thrombolysis for superior vena cava syndrome post bone marrow transplant: a paediatric experience.

Our case highlights timely diagnosis and management of pulmonary embolism (PE) and superior vena cava syndrome in a child with a background of Chediak-Higashi syndrome who underwent a bone marrow transplant, the probable precipitant being concomitant autoimmune haemolytic anaemia. He was successfully managed in paediatric critical care with a catheter-directed tissue-type Plasminogen activator (tPA) thrombolysis without any complications. He is currently 3 years post transplant with good immune reconstitution.

Mixed T-Cell Chimerism Following Hematopoietic Cell Transplantation for Non-Malignant Disorders Is Common, Facilitates Anti-Viral Immunity, and Is Not Associated with Graft Failure in Pediatric Patients.

Myeloid chimerism better reflects donor stem cell engraftment than whole-blood chimerism in assessing graft function following allogeneic hematopoietic stem cell transplant (HCT). We describe our experience with 130 patients aged younger than 18 years, treated with allogeneic HCT using bone marrow or PBSC from HLA-matched donors for non-malignant diseases, whose pre-transplant conditioning therapy included alemtuzumab and who were monitored with lineage-specific chimerism after transplant. At 6 years post-transplant, overall survival (OS) was 91.

Granulocyte transfusion during cord blood transplant for relapsed, refractory AML is associated with massive CD8 T-cell expansion, significant cytokine release syndrome and induction of disease remission.

In high-risk myeloid malignancy, relapse is reduced using cord blood transplant (CBT) but remains the principal cause of treatment failure. We previously described T-cell expansion in CBT recipients receiving granulocyte transfusions. We now report the safety and tolerability of such transfusions, T-cell expansion data, immunophenotype, cytokine profiles and clinical response in children with post-transplant relapsed acute leukaemia who received T-replete, HLA-mismatched CBT and pooled granulocytes within a phase I/II trial (ClinicalTrials.