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  • ||||||||||  Melblez Kit (melphalan hepatic delivery system) / Delcath
    Trial completion date, Trial primary completion date:  FOCUS: Percutaneous Hepatic Perfusion in Patients With Hepatic-dominant Ocular Melanoma (clinicaltrials.gov) -  Apr 3, 2023   
    P3,  N=102, Active, not recruiting, 
    Not yet recruiting --> Recruiting Trial completion date: May 2023 --> May 2025 | Trial primary completion date: Dec 2022 --> May 2023
  • ||||||||||  oNKord (inaleucel) / Glycostem, Medac, Korea Kolmar
    Trial primary completion date, Combination therapy, IO biomarker:  NK4AML: Natural Killer-cell Therapy for Acute Myeloid Leukemia (clinicaltrials.gov) -  Mar 29, 2023   
    P1/2,  N=23, Recruiting, 
    Trial completion date: May 2023 --> May 2025 | Trial primary completion date: Dec 2022 --> May 2023 Trial primary completion date: Jun 2023 --> Sep 2023
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    Biomarker, Trial completion date, Trial primary completion date:  Muscle Dysfunction in Patients With Hematological Diseases Referred to Stem Cell Transplant (clinicaltrials.gov) -  Mar 29, 2023   
    P=N/A,  N=144, Not yet recruiting, 
    Trial primary completion date: Jun 2023 --> Sep 2023 Trial completion date: Dec 2022 --> Dec 2026 | Trial primary completion date: Dec 2022 --> Dec 2026
  • ||||||||||  Hematopoietic Stem Cell Transplantation in rheumatology (South Hall, Session room 2) -  Mar 18, 2023 - Abstract #EULAR2023EULAR_1184;    
    During the neonatal period, she developed fever associated with an increase of inflammatory parameters, respiratory failure, cutaneous rash, hepatosplenomegly and growth failure. Based on clinical suspicion of MA, urinary mevalonic acid was quantified and was shown to be markedly increased (7024 mmol/mmol creatinine, n.v. 3000 mmol/mmol creatinine).
  • ||||||||||  Melblez Kit (melphalan hepatic delivery system) / Delcath
    Trial primary completion date:  FOCUS: Percutaneous Hepatic Perfusion in Patients With Hepatic-dominant Ocular Melanoma (clinicaltrials.gov) -  Mar 7, 2023   
    P3,  N=102, Active, not recruiting, 
    Low-dose lymphoid irradiation in suspected immune-mediated GF may be a feasible option. Trial primary completion date: Jun 2022 --> Dec 2022
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    OUTCOMES OF BUSULFAN VERSUS TREOSULFAN BASED REGIMENS FOLLOWED BY AUTOLOGOUS STEM CELL TRANSPLANT IN CHILDREN WITH HIGH RISK EWING SARCOMA (ePoster Area) -  Feb 11, 2023 - Abstract #EBMT2023EBMT_1890;    
    The high dose regimens used consist of either busulfan and melphalan or treosulfan and melphalan followed by autologous stem cell rescue (ASCR).The goal of our study was to compare the efficacy and toxicity of both regimens. Our single center cohort showed no statistically significant differences in OS and RFS between the BBR group and the TBR group, mostly due to small cohort size.Both regimens were relatively safe as shown by low incidence of major complications (i.e. no events of VOD) with statistically significant lower incidence of mucositis in the TBR group.A larger cohort, using larger, multicenter database is required to better explore the difference in outcomes and toxicity between BBR and TBR.
  • ||||||||||  Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi
    HAPLOIDENTICAL STEM CELL TRANSPLANTATION WITH EX-VIVO T-DEPLETION IN PRIMARY IMMUNODEFICIENCY AND IMMUNE REGULATORY SYNDROMES: A MULTICENTER EXPERIENCE (ePoster Area) -  Feb 11, 2023 - Abstract #EBMT2023EBMT_1807;    
    Our single center cohort showed no statistically significant differences in OS and RFS between the BBR group and the TBR group, mostly due to small cohort size.Both regimens were relatively safe as shown by low incidence of major complications (i.e. no events of VOD) with statistically significant lower incidence of mucositis in the TBR group.A larger cohort, using larger, multicenter database is required to better explore the difference in outcomes and toxicity between BBR and TBR. Characteristics of the 48 haplo-HSCTsCharacteristicsn%DiagnosisPIDD #PIRD ##3216 66.733.3HaploplatformTCR ab+/CD19+ negative selectionCD3+/CD19+ negative selectionCD34+ positive selection34131 70.827.12.1Karnofsky/Lansky at Haplo 60741 14.685.4Conditioning regimensFlu-based*Bus-basedTreo-based**No conditioning194205 39.68.341.710.4RadiotherapyYesTLI / 4 GyTBI 4 GyNo86240 16.7 83.3SerotherapyATG
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus, Defitelio (defibrotide) / IRCCS San Raffaele Hospital, Jazz
    UNDERGOING TRANSPLANT FOR MALIGNANT OSTEOPETROSIS: DON (ePoster Area) -  Feb 11, 2023 - Abstract #EBMT2023EBMT_1804;    
    Characteristics of the 48 haplo-HSCTsCharacteristicsn%DiagnosisPIDD #PIRD ##3216 66.733.3HaploplatformTCR ab+/CD19+ negative selectionCD3+/CD19+ negative selectionCD34+ positive selection34131 70.827.12.1Karnofsky/Lansky at Haplo 60741 14.685.4Conditioning regimensFlu-based*Bus-basedTreo-based**No conditioning194205 39.68.341.710.4RadiotherapyYesTLI / 4 GyTBI 4 GyNo86240 16.7 83.3SerotherapyATG Thiotepa-busulfan-fludarabine conditioning regimen was used...This time treosulfan
  • ||||||||||  cyclophosphamide / Generic mfg.
    HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR PEDIATRIC SICKLE CELL DISEASE WITHIN THE POST-TRANSPLANT CYCLOPHOSPHAMIDE ERA (ePoster Area) -  Feb 11, 2023 - Abstract #EBMT2023EBMT_1791;    
    We here report our experience with the PT-CY approach in mismatched related and unrelated donor HSCT in pediatric SCD patients treated with a fludarabine-treosulfan-thiotepa -reduced toxicity- conditioning regimen in combination with individualized ATG. We conclude that in the absence of an HLA-identical family or a matched unrelated donor, HSCT by using a mismatched unrelated or haploidentical related donor in the context of individualized ATG plus PT-CY can be considered as an equally safe and effective treatment option for pediatric SCD patients.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus, Campath (alemtuzumab) / Sanofi
    CASE REVIEW OF NUTRITION MANAGEMENT FOR WOLMAN DISEASE DURING ADMISSION FOR HSCT (ePoster Area) -  Feb 11, 2023 - Abstract #EBMT2023EBMT_1704;    
    Subgroup sensitivity analysis did not reveal significant interaction between conditioning intensity and pre-transplant MRD status in terms of transplant outcomes. An 8 month old male with Wolman Disease and secondary HLH was admitted to The Children
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus, Mozobil (plerixafor) / Sanofi
    PEDIATRIC AUTOLOGOUS STEM CELL TRANSPLANTS: A SINGLE CENTRE EXPERIENCE (ePoster Area) -  Feb 11, 2023 - Abstract #EBMT2023EBMT_1703;    
    BEAM conditioning protocol was the standard protocol used in all cases of relapse/refractory lymphoma except 1 patient in whom TBC (Thiotepa, Busulfan, Cyclophosphamide) protocol was used in view of CNS Lymphoma. Busulfan + Cyclophosphamide was used for patients with Relapsed APML, Carboplatin + Etoposide was used for patient with metastatic choriocarcinoma and Treosulphan + Melphalan was used for patient with Ewing
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus, Campath (alemtuzumab) / Sanofi, Vistide (cidofovir) / Gilead
    GRAFT FAILURE COMPLICATING ADENOVIRUS INFECTION: A UK NATIONAL EXPERIENCE (ePoster Area) -  Feb 11, 2023 - Abstract #EBMT2023EBMT_1700;    
    Graft failure associated with invasive adenovirus is a significant problem in paediatric HSCT, especially in the T-cell depleted setting. Long term survival is possible with intensive antiviral therapy and with donor CD34 selected stem cells top up.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    OUTCOME OF TREOSULFAN-BASED CONDITIONING IN 93 INFANTS WITH SCID  (ePoster Area) -  Feb 11, 2023 - Abstract #EBMT2023EBMT_1668;    
    Further prospective randomized comparison to standard-dose chemotherapy, especially at relapse, is required. Ninety (97%) received treosulfan