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  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus, Rituxan (rituximab) / Roche
    HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR C1Q DEFICIENCY (CARRON) -  Feb 14, 2024 - Abstract #EBMT2024EBMT_1664;    
    This generates an extra challenge when counselling patients with a positive family history. HSCT, especially if performed in the early course of the disease, may be a valid and definitive treatment.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    HEMATOPOETIC STEM CELL TRANSPLANTATION WITH CURRENT PERSPECTIVE IS SAFE FOR MUCOPOLYSACCHARIDOSIS TYPE VI PATIENTS (CLYDE) -  Feb 14, 2024 - Abstract #EBMT2024EBMT_1656;    
    However, as all our patients were alive on the last follow-up, high complications such as GVHD and mortality in previous studies were not observed in our study, and adequate enzyme levels were achieved without enzyme replacement therapy after transplantation. The walking tests of all evaluable patients showed significant improvements over pre-HSCT results.Our conditioning regimen, which did not consist of cyclophosphamide and TBI, contrary to previous reports, is a promising regimen that has a very low toxicity profile.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    HEMATOPOETIC STEM CELL TRANSPLANTATION WITH CURRENT PERSPECTIVE IS SAFE FOR MUCOPOLYSACCHARIDOSIS TYPE VI PATIENTS (ePoster area) -  Feb 14, 2024 - Abstract #EBMT2024EBMT_1655;    
    However, as all our patients were alive on the last follow-up, high complications such as GVHD and mortality in previous studies were not observed in our study, and adequate enzyme levels were achieved without enzyme replacement therapy after transplantation. The walking tests of all evaluable patients showed significant improvements over pre-HSCT results.Our conditioning regimen, which did not consist of cyclophosphamide and TBI, contrary to previous reports, is a promising regimen that has a very low toxicity profile.
  • ||||||||||  HAEMATOPOIETIC STEM CELL TRANSPLANTATION FOR CTLA-4 INSUFFICIENCY: AN EBMT INBORN ERRORS WORKING PARTY STUDY (LOMOND) -  Feb 14, 2024 - Abstract #EBMT2024EBMT_1612;    
    Patients received predominantly treosulfan-based conditioning (n=21, 60%, with thiotepa: n=12)...Five patients received post-transplant cyclophosphamide.Four-year OS was 93.8% (77.4 We report the largest cohort to date and examine the impact of pre-HSCT CTLA-4-Ig therapy (abatacept, belatacept) and degree of pre-HSCT immune dysregulation on survival and immunological outcome...Pre-HSCT, 24/40 (60%) received CTLA-4-Ig as well as other immunomodulatory therapies (including glucocorticoids, n=34, 85%; rituximab, n=20, 50%)...Conditioning was predominantly treosulfan-based (n=30, 75%, Table 1) with serotherapy (alemtuzumab: 21/40, ATG: 16/40).Median follow-up was 3 years (0.5
  • ||||||||||  cyclophosphamide / Generic mfg.
    ENGRAFTMENT SYNDROME AND ACUTE GVHD AFTER HAPLOIDENTICAL POST-TRANSPLANT CYCLOPHOSPHAMIDE: A PAEDIATRIC MULTICENTRIC EXPERIENCE (CLYDE) -  Feb 14, 2024 - Abstract #EBMT2024EBMT_1435;    
    day 0 (0; -1) in 20 patients, and it was started > day 0 in 79 patients (+5 in 77; +6 in 1; and +1 in 1)... ES is a severe complication of HSCT, its incidence appears to be associated with use of PBSC and with hight count of mononuclear cells engrafted, but the timing of initiation of CNI does not seem to affect its incidence, while an earlier initiation of CNI appears to be associated with development of a-GVHD.
  • ||||||||||  cyclophosphamide / Generic mfg.
    ENGRAFTMENT SYNDROME AND ACUTE GVHD AFTER HAPLOIDENTICAL POST-TRANSPLANT CYCLOPHOSPHAMIDE: A PAEDIATRIC MULTICENTRIC EXPERIENCE (ePoster area) -  Feb 14, 2024 - Abstract #EBMT2024EBMT_1434;    
    day 0 (0; -1) in 20 patients, and it was started > day 0 in 79 patients (+5 in 77; +6 in 1; and +1 in 1)... ES is a severe complication of HSCT, its incidence appears to be associated with use of PBSC and with hight count of mononuclear cells engrafted, but the timing of initiation of CNI does not seem to affect its incidence, while an earlier initiation of CNI appears to be associated with development of a-GVHD.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus, xevinapant (Debio 1143) / EMD Serono, Rituxan (rituximab) / Roche
    EARLY ONSET OF EPSTEIN-BARR VIRUS POSITIVE MULTIPLE MYELOMA TYPE OF POSTTRANSPLANT LYMPHOPROLIFERATIVE DISORDER  (ePoster area) -  Feb 14, 2024 - Abstract #EBMT2024EBMT_1361;    
    Available data suggest that monomorphic multiple myeloma PTLD tends to behave in manner more similar to lymphomas with mass leasions rather than bone marrow impairment. In this case two related histopatology results indicate highly-agrresive type of disease and unfavourable clinical course
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus, Rituxan (rituximab) / Roche
    DONOR-DERIVED IMMUNE RECONSTITUTION PROFILE OF AGGRESSIVE NEUROMYELITIS OPTICA IN LONG-TERM DISEASE REMISSION AFTER ALLOGENEIC TRANSPLANTATION (Hall 3 North) -  Feb 14, 2024 - Abstract #EBMT2024EBMT_1340;    
    The effectiveness of allogeneic HSCT in achieving sustained remission in NMO patients is noteworthy. Our immunological results suggest that allogeneic HSCT can provide long-term disease control in refractory NMO through several concurring mechanisms: the eradication of autoreactive cell clones by high-dose chemotherapy and by in vivo T and B cell depletion, elimination of long-lived plasma cells producing AQP4-Ab, the re-establishment of thymic central tolerance and renewal of the immune repertoire.
  • ||||||||||  DONOR-DERIVED CAR-T CELLS ARE SAFELY AND EFFECTIVELY CO-INFUSED WITH THE ALLOGENIC GRAFT, DEPLETED OF AB T CELLS IN CHILDREN WITH ADVANCED B-CELL NEOPLASM (Hall 3 South) -  Feb 14, 2024 - Abstract #EBMT2024EBMT_1334;    
    Our immunological results suggest that allogeneic HSCT can provide long-term disease control in refractory NMO through several concurring mechanisms: the eradication of autoreactive cell clones by high-dose chemotherapy and by in vivo T and B cell depletion, elimination of long-lived plasma cells producing AQP4-Ab, the re-establishment of thymic central tolerance and renewal of the immune repertoire. All patients had relapse after multiple lines of treatment including previous HSCT(n=10), blinatumomab (n=12), inotuzumab (n=1) and CAR-T cell infusion(n=8)...PBMS used to produce CAR T cells were provided by the patient
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus, Orencia (abatacept) / BMS, Valcyte (valganciclovir) / Roche, Mitsubishi Tanabe
    CONTROVERSIAL INDICATION TO TRASPLANT: NOW OR NEVER? (ePoster area) -  Feb 14, 2024 - Abstract #EBMT2024EBMT_1233;    
    He presented CMV reactivation 30 days after transplant successfully treated with Valganciclovir... In the context of primary immunodeficiencies and immune-dysregulatory diseases, partial responses to conventional treatments together with the risk of increasing the inflammatory burden or favouring the development of colonizations that can expose patients to major complications, suggest that the timing of transplant can be crucial.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    CONDITIONING FOR SECOND HSCTS FROM THE SAME DONOR AFTER MYELOID AUTOLOGOUS RECONSTITUTION IN PATIENTS WITH IEI AND THALASSEMIA: IMMUNOSUPPRESSION IS NOT ALWAYS REQUIRED (CLYDE) -  Feb 14, 2024 - Abstract #EBMT2024EBMT_1220;    
    Patients received a conditioning regimen containing predominantly myelosuppressive drugs (Treosulfan n=4, Treosulfan/Thiotepa n=2, Busulfan n=2, Busulfan/Melphalan n=1) or radioimmunotherapy (n=2) avoiding immunosuppression or serotherapy (except in one patient with autoimmune phenomena before his 2nd HSCT) before the administration of T-cell replete (n=4), T-cell reduced (n=2) or T-cell depleted (n=5) grafts from the same donor. In summary these data demonstrate that for this cohort a selective myelosuppressive conditioning regimen for a second transplant from the same donor after autologous myeloid reconstitution is a feasible approach to achieve stable myeloid engraftment with low toxicity, treatment related morbidity/ mortality, or infectious complications.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    CONDITIONING FOR SECOND HSCTS FROM THE SAME DONOR AFTER MYELOID AUTOLOGOUS RECONSTITUTION IN PATIENTS WITH IEI AND THALASSEMIA: IMMUNOSUPPRESSION IS NOT ALWAYS REQUIRED (ePoster area) -  Feb 14, 2024 - Abstract #EBMT2024EBMT_1219;    
    Patients received a conditioning regimen containing predominantly myelosuppressive drugs (Treosulfan n=4, Treosulfan/Thiotepa n=2, Busulfan n=2, Busulfan/Melphalan n=1) or radioimmunotherapy (n=2) avoiding immunosuppression or serotherapy (except in one patient with autoimmune phenomena before his 2nd HSCT) before the administration of T-cell replete (n=4), T-cell reduced (n=2) or T-cell depleted (n=5) grafts from the same donor. In summary these data demonstrate that for this cohort a selective myelosuppressive conditioning regimen for a second transplant from the same donor after autologous myeloid reconstitution is a feasible approach to achieve stable myeloid engraftment with low toxicity, treatment related morbidity/ mortality, or infectious complications.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (HSCT) TO CHILDREN WITH PRIMARY IMMUNODEFICIENCY  (ePoster area) -  Feb 14, 2024 - Abstract #EBMT2024EBMT_905;    
    Transplantation offers cure to the majority of patients with PID. As Treosulfan was associated with higher possibility of rejection in non-SCID whereas, Busulfan had higher toxicity, a tailored approach should be adopted for the selection of the conditioning in each particular variant of PID.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    ACUTE GVHD IN PATIENTS ENROLLED IN THE PROSPECTIVE, INTERNATIONAL FORUM TRIAL: CLINICAL RESULTS (LOMOND) -  Feb 14, 2024 - Abstract #EBMT2024EBMT_861;    
    P2/3
    Moreover, we observed that a higher grade of aGvHD correlated with an increased risk of developing cGvHD. Given the association in univariate analysis of grade III-IV aGvHD and 9/10 matched HSCT, not paralleled by a reduction of disease relapse, more efficacious strategies for GvHD prophylaxis strategies are warranted in patients transplanted with a donor other than HLA fully matched
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    5-YEAR TRANSPLANT SUCCESS AFTER TREOSULFAN CONDITIONING (CLYDE) -  Feb 14, 2024 - Abstract #EBMT2024EBMT_793;    
    The return-to-work status in our population is consistent with reports from the registry studies [Bhatt et al. JTCT 2021;27(8):679.e1-679.e8], while GRFS and CRFS appear to be superior to what has been reported in large registry studies with other regimens [Mehta et al.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    5-YEAR TRANSPLANT SUCCESS AFTER TREOSULFAN CONDITIONING (ePoster area) -  Feb 14, 2024 - Abstract #EBMT2024EBMT_792;    
    The return-to-work status in our population is consistent with reports from the registry studies [Bhatt et al. JTCT 2021;27(8):679.e1-679.e8], while GRFS and CRFS appear to be superior to what has been reported in large registry studies with other regimens [Mehta et al.
  • ||||||||||  oNKord (inaleucel) / Glycostem, Medac, Korea Kolmar
    Preclinical, Journal:  Qualification of a flow cytometry-based method for the evaluation of in vitro cytotoxicity of GTA002 natural killer cell therapy. (Pubmed Central) -  Feb 2, 2024   
    P1/2
    Notably, we identified relevant aspects to address when progressing towards method validation to support pivotal clinical studies. This article provides a "case-study" of how analytical method development for cell therapeutics is planned and executed from early clinical stages, anticipating the need to establish robust procedures to overcome scientific and regulatory challenges during method validation.
  • ||||||||||  Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi
    Enrollment change, Trial completion date, Trial primary completion date:  FORUM: Allogeneic Stem Cell Transplantation for Children and Adolescents With Acute Lymphoblastic Leukaemia (clinicaltrials.gov) -  Jan 16, 2024   
    P2/3,  N=1800, Recruiting, 
    To sum up, our findings strongly suggest that clinical application of TBSM1 may serve as a vasoactive drug treatment to suppress tumor progression. N=1000 --> 1800 | Trial completion date: Apr 2026 --> Apr 2030 | Trial primary completion date: Apr 2021 --> Jun 2025