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Novel treatment of severe combined immunodeficiency utilizing ex-vivo T-cell depleted haploidentical hematopoietic stem cell transplantation and CD45RA+ depleted donor lymphocyte infusions.

BACKGROUND: Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment available for severe combined immunodeficiency (SCID); although, there is a high incidence of severe infections and an increased risk of graft-versus host-disease (GvHD) with HSCT. Early intervention is a crucial prognostic factor and a HLA-haploidentical parental donor is often available. Haploidentical HSCT protocols utilizing extensively ex vivo T-cell depleted grafts (CliniMACs system) have proven efficient in preventing GvHD, but cause a delay in early T-cell recovery that increases the ri…

Gamma delta T cell reconstitution is associated with fewer infections and improved event-free survival after hematopoietic stem cell transplantation for pediatric leukemia.

After hematopoietic stem cell transplantation (HSCT), successful engraftment and immune recovery is necessary to protect the patient from relapse and infection. Many studies highlight the importance of conventional αβ T cell recovery after HSCT, but the impact of γδ T cell recovery has not been well described. Here, we investigate the recovery of γδ T cells in 102 pediatric patients with acute leukemia in first clinical remission who underwent allogeneic HSCT at St. Jude Children's Research Hospital from 1996 to 2011. Mean patient age was 10.5 ± 5.9 years (range, .6 to 25.2), and mean survivor…

Transplantation of human embryonic stem cell-derived neural precursor cells and enriched environment after cortical stroke in rats: cell survival and functional recovery.

Cortical stem cell transplantation may help replace lost brain cells after stroke and improve the functional outcome. In this study, we transplanted human embryonic stem cell (hESC)-derived neural precursor cells (hNPCs) or vehicle into the cortex of rats after permanent distal middle cerebral artery occlusion (dMCAO) or sham-operation, and followed functional recovery in the cylinder and staircase tests. The hNPCs were examined prior to transplantation, and they expressed neuroectodermal markers but not markers for undifferentiated hESCs or non-neural cells. The rats were housed in either enr…

Effect of CD34 cell dose on hematopoietic reconstitution and outcome in 508 patients with multiple myeloma undergoing autologous peripheral blood stem cell transplantation.

BACKGROUND: We analyzed the hematopoietic reconstitution and outcome of 508 patients with multiple myeloma (MM) with respect to the number of CD34+ cells reinfused at our center. PATIENTS AND METHODS: Each cohort of 390 patients (unselected CD34+ cell transplant) and 118 patients (CD34+ selected transplant) was divided into four subgroups. Among the 390 transplantations, 86 patients received a high dose (HD-) of > or =6.50 x 10(6) unselected CD34+ cells/kg, 116 patients a low dose (LD-) of <3.00 x 10(6) CD34+ cells/kg. Among the patients treated with CD34+ selected PBSC, 34 received > or =6.5

[Cyclosporin A withdrawal causes spontaneous remission of recurrent subcutaneous tumors after allogeneic peripheral blood stem cell transplantation for adult T-cell leukemia/lymphoma].

A 36-year-old woman was referred to our hospital because of leukocytosis in June 2000, and was admitted to our hospital and diagnosed as having adult T-cell leukemia/lymphoma (ATL; acute type). Complete remission was achieved with eight courses of CHOP therapy, but ATL relapsed and she was readmitted to our hospital in September 2001. Laboratory examination showed elevated levels of serum LDH and soluble IL-2 receptor, and hypercalcemia. CT examinations showed swelling of the abdominal lymph nodes and hepatosplenomegaly. CHOP therapy improved the symptoms, but recrudescence soon occurred. Afte

Haematopoietic stem cell transplantation outcomes for teenage and young adult patients with acute leukaemia: A British Society of Blood and Marrow Transplantation and Cellular Therapy registry study.

Teenage and young adult (TYA) patients undergoing allogeneic stem cell transplant have distinct psychosocial needs, yet they are poorly represented in research and their outcomes are not well understood. This study uses prospectively collected data from the British Society of Blood and Marrow Transplantation and Cellular Therapy (BSBMTCT) registry to explore UK transplant practice and outcomes for TYA patients (aged 16-24) in this healthcare setting, alongside children (aged 1-15) and adults (aged 25-39), transplanted for acute leukaemia (including lymphoblastic, acute lymphoblastic leukaemia

Defining remission and relapse after allogeneic hematopoietic cell transplantation in myelodysplastic/myeloproliferative neoplasms and optimization of transplantation outcomes: Recommendations from the EBMT practice harmonisation and guidelines committee.

Myelodysplastic/myeloproliferative neoplasms (excluding CMML) are rare and heterogenous malignancies with recurrent and overlapping clinicopathological abnormalities. Classification and characterization continue to evolve, but outcomes remain unsatisfactory and allo-HCT is currently the only curative therapy. Experience with these diseases is limited by rarity, accounting for <1% of allo-HCT procedures in Europe annually. Transplant indications have recently been described by this group, however response assessment, post-transplant surveillance and management of poor graft function, splenomega

Duration of Hospitalization is Associated with the Gut Microbiome in Patients Undergoing Hematopoietic Stem Cell Transplantation: Early Results from a Randomized Trial of Home Versus Hospital Transplantation.

Home-based hematopoietic stem cell transplantation (HCT) is an innovative care model with growing interest, but its impact on the gut microbiome remains unexplored in a randomized setting. We present interim results from the first randomized controlled trials (RCT) evaluating the effect of HCT location-home versus hospital-on gut microbial diversity and antimicrobial resistance (AMR) gene carriage. We hypothesize that patients randomized to undergo home HCT would have higher gut taxonomic diversity and lower AMR gene abundance compared to those undergoing standard hospital HCT. We analyzed sto…

Global characteristics and outcomes of autologous hematopoietic stem cell transplantation for newly diagnosed multiple myeloma: A study of the worldwide network for blood and marrow transplantation (WBMT).

Autologous hematopoietic cell transplantation (AHCT) is a commonly used treatment in multiple myeloma (MM). However, real-world global demographic and outcome data are scarce. We collected data on baseline characteristics and outcomes from 61 725 patients with newly diagnosed MM who underwent upfront AHCT between 2013 and 2017 from nine national/international registries. The primary endpoint was overall survival (OS), and the secondary endpoints were progression-free survival (PFS), relapse incidence (RI) and non-relapse mortality (NRM). Median OS amounted to 90.2 months (95% CI 88.2-93.6) and…

Subcutaneous transplantation of human thyroid tissue into a pre-vascularized Cell Pouch™ device in a Mus musculus model: Evidence of viability and function for thyroid transplantation.

This study aimed to investigate the survival and efficacy indicators of human thyroid tissue transplantation into a retrievable, prevascularized implanted Sernova Corp Cell Pouch™ (CP) device. Thyroid tissue from human donors was transplanted subcutaneously into the pre-implanted CP device or into the subcutaneous (SC) space alone as a control in a nude Mus musculus model. Transplanted M. musculus were monitored for human serum thyroglobulin (TG) levels for 3 months until the transplants were removed for histological assessment. Human thyroid tissue survived and continued to produce TG in tran

Prophylaxis and management of graft versus host disease after stem-cell transplantation for haematological malignancies: updated consensus recommendations of the European Society for Blood and Marrow Transplantation.

Graft-versus-host disease (GVHD) is a major factor contributing to mortality and morbidity after allogeneic stem-cell transplantation. Because of the small number of results from well designed, large-scale, clinical studies there is considerable variability in the prevention and treatment of GVHD worldwide. In 2014, to standardise treatment approaches the European Society of Blood and Marrow Transplantation published recommendations on the management of GVHD in the setting of HLA-identical sibling or unrelated donor transplantation in adult patients with haematological malignancies. Here we up…

Safety and Efficacy of Haploidentical Peripheral Blood Stem Cell Transplantation for Myeloid Malignancies Using Post-transplantation Cyclophosphamide and Anti-thymocyte Globulin as Graft-versus-Host Disease Prophylaxis.

Haploidentical stem cell transplantation (haploSCT) has greatly improved access to curative treatment for myeloid malignancies in patients without suitable matched sibling/unrelated donors. We investigated the safety and efficacy of haploSCT after reduced intensity conditioning (RIC) with anti-thymocyte globulin (ATG), post-transplant cyclophosphamide (PTCy), and cyclosporine to prevent rejection and graft-versus-host disease (GVHD). In this study, 47 patients received RIC using fludarabine, busulfan, and total body irradiation (200 cGy). Unmanipulated peripheral blood grafts were used. GVHD p…

Association Between the Time of Day of Post-Transplant Cyclophosphamide Administration and Haemorrhagic Cystitis After Allogeneic Haematopoietic Stem Cell Transplantation.

OBJECTIVE: To evaluate the impact of the time-of-day (daytime vs. nighttime) of the infusion of post-transplantation cyclophosphamide (PTCy) on the occurrence of haemorrhagic cystitis (HC) in adult patients undergoing allogeneic haematopoietic stem cell transplantation (allo-HSCT). METHODS: We conducted a retrospective, single-centre cohort study including adult allo-HSCT between January 2014 and December 2022. All patients received PTCy at a dose of 50 mg/kg/day for two consecutive days. Patients were classified according to the timing of the first PTCy dose: daytime (06:00-17:59) or nightti…

Modeling donor age and type trade-offs for personalized selection in allogeneic hematopoietic cell transplantation with post-transplant cyclophosphamide prophylaxis.

The prevailing fixed-hierarchy approach to donor selection for allogeneic hematopoietic cell transplantation (HCT) fails to capture critical interactions between donor age and type. We addressed this by analyzing 1713 adult recipients of matched related (MRD), matched unrelated (MUD), or haploidentical donors, receiving post-transplantation cyclophosphamide, using both regularized-Cox and XGBoost machine learning models. Our analysis revealed that the overall, independent association of donor age with survival was modest; however, its importance became pivotal within specific, context-dependen…

CHIME: a novel HLA eplet model associates with non-relapse mortality in haploidentical hematopoietic cell transplant with post-transplant cyclophosphamide.

The importance of HLA mismatch in haploidentical hematopoietic cell transplantation (HaploHCT) with post-transplant cyclophosphamide (PTCy) is debated. If HLA mismatch does impact outcomes, a molecular mismatch model could predict transplant outcomes more effectively than allele mismatch. In this retrospective study, we quantified molecular HLA disparity using eplet mismatch for 265 patient-donor pairs undergoing HaploHCT with PTCy for hematologic malignancy. We discovered an interaction among eplet mismatch (EpMM), HLA class, and mismatch vector that was associated with clinical outcomes. Thi…

Pre- and post-transplant serum albumin levels and outcomes after allogeneic hematopoietic cell transplantation for adults with acute myeloid leukemia.

Hypoalbuminemia has been repeatedly linked to increased non-relapse mortality (NRM) after allogeneic hematopoietic cell transplantation (HCT); a recent analysis also suggested an association with relapse risk. To evaluate the relationship between hypoalbuminemia, NRM, and relapse, we assessed its prognostic role in 1258 adults with MDS (myelodysplastic neoplasm)/AML or AML allografted while in remission between 2006 and 2023. After multivariable adjustment, low pre-HCT serum albumin levels were statistically significantly associated with NRM (hazard ratio [HR] = 0.67, p = 0.028), relapse-free …

A Rare Case of Plasmacytoma-like Post-transplant Lymphoproliferative Disorder in a Pediatric Hematopoietic Stem Cell Transplant Recipient Treated With Daratumumab.

Post-transplant lymphoproliferative disorder (PTLD) is a complication of immunosuppressive therapy following solid organ transplantation (SOT) and hematopoietic stem cell transplantation (HSCT). Although PLTD usually presents as B-cell proliferation, plasmacytoma-like PTLD, a rare subtype of monomorphic PTLD, has been described, mostly in SOT recipients. Only 2 cases of this disease entity have been previously reported in patients after HSCT. While the treatment of choice for PTLD is the reduction of immunosuppression combined with rituximab (anti-CD20 monoclonal antibody), the optimal treatme…

Haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide in the public Chilean national health system: A single center study.

INTRODUCTION: Haploidentical peripheral stem cell transplantation with post-transplant cyclophosphamide is the most common modality in low-and-middle-income countries. This article reports the consecutive adult patients who received this modality of transplant in a single center in Chile between 2016-2021. METHODS: The primary outcome was overall survival. Secondary outcomes were event-free survival, II-IV acute graft-versus-host disease at Day +100, chronic graft-versus-host disease at two years and cumulative incidence of relapse. RESULTS: The median age was 25 years (Range: 15-51), and 65…

Оценка показателей питания перед трансплантацией в качестве прогнозов пневмонии и смертности у многих пациентов с миеломой после аутологической трансплантации стволовых клеток.

Фон/aimPneumonia является существенной причиной заболеваемости и смертности в течение первого года после аналоговой трансплантации стволовых клеток у пациентов с множественной миеломой (ММ), и состояние питания может влиять на осложнения после трансплантации. В нашем исследовании мы стремились изучить взаимосвязь между гериатрическим индексом питания (ГНРИ), прогнозным индексом питания (ПНИ) и счетом КОНУТ, а также частотностью пневмонии и смертностью внутри страны. 1 год после трансплантации 286 пациентов, прошедших аутологическую трансплантацию стволовых клеток (ASCT) в дополнение к диагност

Совпадение в аннотации оригинала
Background/aimPneumonia is a significant cause of morbidity and mortality within the first year after autologous hematopoietic stem cell transplantation in patients with multiple myeloma (MM), and nutritional status may influence complications after transplantation. In our study, we aimed to investigate the relationship between geriatric nutritional index (G…