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- Ovastat (treosulfan) / Medac, Medexus
A RETROSPECTIVE STUDY ANALYZING THE OUTCOME OF HEMATOPOIETIC STEM CELL TRANSPLANTATION IN CHILDREN AND ADOLESCENTS WITH THERAPY-RELATED MYELODYSPLASTIC SYNDROME (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1652;
Cytogenetics and gene mutation profiles had the strongest impact on outcome. Characterization of the genetic profile, including the identification of germline variants, will be required for informed treatment decisions and will help elucidate the pathogenesis of t-MDS.
- Ovastat (treosulfan) / Medac, Medexus
TREOSULFAN BASED CONDITIONING FOR HAEMATOPOIETIC STEM CELL TRANSPLANTATION (HSCT) IN CHILDREN: ON BEHALF OF THE UK PAEDIATRIC BMT GROUP (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1650;
This large, multicentre, multi-disease cohort shows that treosulfan based conditioning offers good overall and event-free survival in children that require HSCT for a variety of conditions. The addition of thiotepa does not affect overall survival or TRM.
- Ovastat (treosulfan) / Medac, Medexus
EXPERIENCE WITH FLUDRABINE/TREOSULFAN/ANTITHYMOCYTEGLOBULIN(FLU/TREO/ATG) CONDITIONING FOR MATCHED DONOR TRANSPLANT IN MYELOID MALIGNANCIES (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1620;
This single-center analysis demonstrates that allo-SCT with Flu/Treo/ATG conditioning is feasible with acceptable TRM. However, a longer follow-up inspection is required to determine its aptness for deep-rooted control of the underlying disease.
- Ovastat (treosulfan) / Medac, Medexus
WOULD MONITORIZING TREOSULFAN LEVELS IN PATIENTS TRANSPLANTED FOR TRANSFUSION DEPENDENT THALASSEMIA BE BENEFICIAL IN TERMS OF CHIMERIZM? A SINGLE CENTER EXPERIENCE (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1615;
There are few studies which correlates treosulfan AUC level with posttransplant consequences in pediatric age group. Although there are publications state that no correlation is needed for treosulfan, expanding patient group and repeating study for follow up in terms of posttransplant toxicity and long term consequences will be beneficial.
- Ovastat (treosulfan) / Medac, Medexus
LONG-TERM SAFETY AND EFFICACY OF REDUCED TOXICITY CONDITIONING REGIMENS IN OLDER RECIPIENTS OF ALLOGENEIC HEMATOPOIETIC CELL TRANSPLANTATION FOR ACUTE LEUKEMIAS (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1614;
Our real-world study confirms that alloHCT with reduced toxicity regimens is feasible in older patients with acute leukemias. The choice of alloHCT in this special patient population of a rather older age needs to be personalized.
- Ovastat (treosulfan) / Medac, Medexus
THE IMPACT OF TREOSULFAN -BASED CONDITIONING FOR PRIMARY IMMUNE DEFICIENCIES: SINGLE CENTER EXPERIENCE (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1612;
Like the previous literature, we did not find any significant change in toxicities or outcomes related to HSCT according to AUCs. Although the efficacy of 12gr/m2 and 14 gr/m2 doses is the same, mixed chimerism rates are higher in the 14 gr/m2 group.
- Ovastat (treosulfan) / Medac, Medexus
PREVIOUS BUSULFAN EXPOSURE IS NOT A CONTRAINDICATION FOR SECOND ALLOGRAFT WITH REDUCED INTENSITY TREOSULFAN BASED CONDITIONING FOLLOWED BY IN-VIVO T-CELL DEPLETED UNRELATED DONOR HAEMATOPOIETIC TRANSPLANT (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1610;
Outcomes for this high-risk population are favorable with no deaths recorded from TRM, infection or graft failure. While follow-up has been limited, the OS is encouraging and the LFS and incidence of acute GVHD are comparable to recorded rates in the literature.Previous conditioning with Busulfan does not preclude the efficacy of Treosulfan-based conditioning in second HSCT as it seems to offer an excellent anti-leukaemia effect without life threatening toxicities.
- Ovastat (treosulfan) / Medac, Medexus, Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi
THIOTEPA, TREOSULFAN AND FLUADARABNE (TTF) VERSUS THIOTEPA, BUSULFAN AND FLUDARABINE (TBF) CONDITIONING FOR MATCHED RELATED DONOR PERIPHERAL BLOOD STEM CELL TRANSPLANT FOR THALASSEMIA MAJOR (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1606;
Background: Traditionally used busulfan (BU) and cyclophosphamide (Cy) based conditioning for Haemopoietic Stem Cell Transplantation for Thalassemia Major is associated with higher veno-occlusive diseases (VOD) and mortality...Total doses were-Fludarabine-160mg/m2, Thiotepa-10mg/kg and Rabbit ATG(Thymoglobulin)- 4.5mg/kg...Graft vs. host disease (GVHD) prophylaxis was with cyclosporine and methotrexate ( Day+1,3,6 and 11)... Both TBF and TTF conditioning regimens are safe and effective for children with thalassemia major undergoing MRD PBSCT.
- Ovastat (treosulfan) / Medac, Medexus
TREOSULFAN, THIOTEPA AND FLUDARABINE CONDITIONING REGIMEN PRIOR TO ALLOGENEIC STEM CELL TRANSPLANTATION IN ACUTE MYELOID LEUKEMIA AND HIGH-RISK MYELODYSPLASTIC SYNDROMES: A SINGLE CENTER EXPERIENCE (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1605;
Relapse was the main cause of death, hence a higher treosulfan dosing and/or a faster immunosuppression tapering might be considered given the low GVHD incidence. Finally, we observed a high rate of GRFS with this transplant platform, which may deserve further study if confirmed on large cohorts.Table 1.
- Ovastat (treosulfan) / Medac, Medexus
PREVIOUS BUSULFAN IS NOT A CONTRAINDICATION FOR SECOND ALLOGRAFT WITH REDUCED INTENSITY TREOSULFAN-BASED CONDITIONING REGIMEN FOLLOWED BY ALLOGENEIC HAEMATOPOIETIC STEM-CELL TRANSPLANT FROM UNRELATED DONORS (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1601;
Outcomes for this high-risk population are favorable with no deaths recorded from TRM, infection or graft failure.While follow-up has been limited, the OS is encouraging and the LFS and incidence of acute GVHD are comparable to recorded rates in the literature. Previous conditioning with Busulfan does not preclude the efficacy of Treosulfan-based conditioning in second HSCT as it seems to offer an excellent anti-leukaemia effect without life threatening toxicities.
- Ovastat (treosulfan) / Medac, Medexus, Grafalon (rabbit anti-T-lymphocyte globulin) / Mundipharma, NeoPharm
ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION USING REDUCED TOXICITY VS MYELOABLATIVE CONDITIONING FOR THE TREATMENT OF CHILDREN WITH MYELOID MALIGNANCIES (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1600;
There was no improvement even in patients with MDS who received no therapy prior to HSCT. The optimal conditioning regimen for high-risk pediatric myeloid malignancies should be investigated in large prospective clinical trials.
- Mozobil (plerixafor) / Sanofi
RECIPIENT STEM CELL MOBILIZATION WITH PLERIXAFOR IN PRE-HSCT CONDITIONING REGIMEN FOR NON-MALIGNANT CONDITIONS (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1598;
Addition of plerixafor to conditioning regimen led to decreased incidence of GF in WAS (4,1%) and CGD (8,6%). Apparently, high incidence of GF in other patients might be caused by inadequate myeloblation of MIC regimens used in half of the patients.
- Ovastat (treosulfan) / Medac, Medexus
TREOSULFAN VERSUS BUSULFAN-BASED CONDITIONING FOR ALLOGENIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (HSCT) IN PEDIATRIC PATIENTS WITH PRIMARY IMMUNODEFICIENCY (PID): A SINGLE CENTER REPORT (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1597;
In primary immunodeficiencies, Treosulfan appears to be an excellent alternative to Busulfan, presenting fewer long-term side effects with the same efficacy on chimerism. Further studies with a longer follow-up and with a more homogeneous group of patients (in terms of disease and year of transplantation) are needed to confirm these data.
- Ovastat (treosulfan) / Medac, Medexus
TREOSULFAN-BASED CONDITIONING FOR ALLOGENEIC HSCT IN CHILDREN: A CASE SERIES USING PK DATA AND AUC OF TREOSULFAN IN SINGLE CENTER EXPERIENCE - ADANA, TURKEY (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1595;
Further studies with a longer follow-up and with a more homogeneous group of patients (in terms of disease and year of transplantation) are needed to confirm these data. We show that using a treosulfan-based conditioning regimen is a safe treatment option in pediatric HSCT in different patients group, even in those with high-risk clinical features pre-HSCT or those with no HLA-identical family donor.
- Ovastat (treosulfan) / Medac, Medexus
TREOSULFAN BASED CONDITIONING IN PEDIATRIC PATIENTS WITH NON-MALIGNANT HAEMATOLOGICAL INDICATIONS FOR TRANSPLANT IS ASSOCIATED WITH VERY LOW TREATMENT RELATED MORTALITY AND HIGH OVERALL SURVIVAL (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1590;
The poorer outcomes in osteopetrosis are not dissimilar to other cohorts, reflecting the need for timely diagnosis and transplantation. A prospective randomized comparison with busulfan containing regimens is unlikely to happen, but this real world data suggests a superiority over historical busulfan conditioned cohorts.
- Ovastat (treosulfan) / Medac, Medexus
CHIMERISM ANALYSIS POST-HSCT WITH A TREOSULFAN-BASED CONDITIONING REGIMEN FOR PEDIATRIC NONMALIGNANT DISEASES (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1585;
In our cohort, acute GvHD rate was significantly lower in the MC group, suggesting that a state of MC may pose as an advantage in nonmalignant diseases where FD chimerism is not mandatory. Younger age was associated with developing MC, possibly due to differences in chemotherapy bioavailability and metabolism in younger patients.
- Ovastat (treosulfan) / Medac, Medexus
LONG-TERM THYROID DISORDERS AFTER PAEDIATRIC HCT FOR MALIGNANT UNDERLYING DISEASES (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1574;
Importantly, we identified pre-transplant CT as a significant RF for the development of TD, which should be considered for future studies. In contrast to other studies the CI of Treo-associated TD seems remarkable.
- Ovastat (treosulfan) / Medac, Medexus
EFFECT OF BUSULFAN AND TREOSULFAN ON GONADAL FUNCTION AFTER ALLOGENEIC STEM CELL TRANSPLANTATION IN CHILDREN AND ADOLESCENTS WITH NONMALIGNANT DISEASES IS NOT EXPOSURE-DEPENDENT (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1559;
In contrast to other studies the CI of Treo-associated TD seems remarkable. Our data do not support the premise that reduced intensity BU-based conditioning lowers the risk for gonadal toxicity and it is unlikely that TDM-based reduced treosulfan exposure will further reduce the risk for gonadal dysfunction.
- Ovastat (treosulfan) / Medac, Medexus
TEN-YEAR FOLLOW UP STUDY OF CHILDREN WITH THALASSAEMIA MAJOR POST TRANSPLANTATION USING TREOSULFAN, THIOTEPA, FLUDARABINE BASED CONDITIONING REGIMEN AND ITS IMPACT ON GROWTH AND PUBERTY (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1555;
Our study clearly demonstrates that treosulfan does not have a negative impact on growth and puberty in children with thalassaemia major. Only 6.9 % children required growth hormone supplementation and there was no impact on puberty in both boys and girls with menarche at a median age of 14.8 years Despite the higher cost of treosulfan the reduced late side effects justifies its use in all children undergoing HSCT for thalassaemia major.
- Coversin SC (nomacopan SC) / Akari Therap
CLINICAL RESPONSE TO NOMACOPAN IN THE PAEDIATRIC HSCT-TMA SETTING (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1534;
P3
It is important to be vigilant for clinical signs and symptoms of TMA and screen appropriately when suspicion arises so patients can receive prompt management and complement inhibition considered early. These data show that nomacopan had a favourable safety profile and controlled complement activity in this patient with severe HSCT-TMA.
- Ovastat (treosulfan) / Medac, Medexus
EFFECT OF THE KNOCKOUT OF GSTA1 ON HEPATIC CELLS AND CELLULAR RESPONSE TO DRUGS USED IN HEMATOPOIETIC STEM CELL TRANSPLANTATION (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1501;
In addition, we also demonstrate a correlation between the consequent GSTM1 down-regulation and an increase in GCLC expression resulting in increased GSH levels. This increase leads to a higher cellular resistance to various drugs.
- Ovastat (treosulfan) / Medac, Medexus
WEST-NILE VIRUS MENINGOENCEPHALITIS IN IMMUNOCOMPROMISED HOST AFTER ALLOGENIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1491;
Our case suggests that patient may benefit from the rapid detection of the infection and administration of intravenous immunoglobulins. Due to the long incubation period in immunocompromised hosts, screening for WNV-serology in BMT-recipients should be considered before conditioning, as active infections in immunocompromised hosts has shown to be fatal in most cases and no curative therapy is still available.
- CLINICAL CHARACTERISTICS AND OUTCOMES OF PERI-HSCT COVID-19 INFECTION IN CHILDREN WITH INBORN ERRORS OF IMMUNITY (IEI) (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1485;
In countries where restrictions have been relaxed, leading to relative endemicity of the virus, concomitant SARS-CoV-2 infection in children embarking on HSCT or with significant immunosuppression is expected to become commonplace, highlighting the importance of novel strategies. Our small cohort shows a favourable outcome in paediatric patients with pre-HSCT and persistent COVID-19 infection in children with IEI.
- Orencia (abatacept) / BMS, Entyvio (vedolizumab) / Takeda
POST-TRANSPLANT CYCLOPHOSPHAMIDE (PTCY), ABATACEPT AND VEDOLIZUMAB IN GVHD PROPHYLAXIS IN HEMATOPOIETIC STEM CELLS TRANSPLANTATION FROM MATCHED UNRELATED AND HAPLOIDENTICAL DONORS IN PEDIATRIC ACUTE LEUKEMIA PATIENTS (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1374;
P3
Preliminary data suggest that addition of vedolizumab and abatacept to the PtCy-based GVHD prophylaxis is not associated with increased toxicity and may reduce the incidence of gut GVHD. This approach can be further tested in a prospective trial with the goal to increase the anti-leukemic efficacy of HSCT.
- Ovastat (treosulfan) / Medac, Medexus
SUPERIOR GRAFT-VERSUS-LEUKEMIA EFFECT IN MATCHED UNRELATED DONOR VERSUS HLA-IDENTICAL SIBLING PEDIATRIC RECIPIENTS TRANSPLANTED FOR ACUTE LYMPHOBLASTIC LEUKEMIA WITHIN THE FORUM STUDY (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1343;
P2/3
Our data indicate that the use of MUD is no longer a risk factor in HSCT for childhood ALL. The greater immune-genetic disparity in the MUD/recipient pairs protects from the risk of relapse, while the use of serotherapy prevents the occurrence of cGvHD, with both factors contributing to the observed improved GRFS in patients transplanted from MUD.
- Ovastat (treosulfan) / Medac, Medexus
DONOR FACTORS AFFECTING OUTCOMES OF PTCY BASED T CELL REPLETE HAPLOIDENTICAL TRANSPLANTS: A SINGLE CENTRE EXPERIENCE FROM INDIA (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1328;
The greater immune-genetic disparity in the MUD/recipient pairs protects from the risk of relapse, while the use of serotherapy prevents the occurrence of cGvHD, with both factors contributing to the observed improved GRFS in patients transplanted from MUD. We report outcomes of patients who underwent haploidentical transplants using post-transplant cyclophosphamide (PTCy) and analyze donor factors affecting outcomes...Conditioning regimens used were Flu-Mel140 [22], Flu-Bu (6.4-9.6 mg/kg IV busulfan) [4], Flu-Treosulfan (30 mg/m2 to 42 mg/m2) [24], Flu-TBI (7.2 Gy/8 Gy) [18], Flu-TBI (4 Gy) [1], Flu-TBI (12 Gy) [1] and Flu-Treo-Thiotepa [1]...
- Obnitix (MSC-FFM) / Medac
SARS-COV-2 FAILS TO INFECT MESENCHYMAL STROMAL CELLS (MSCS) AND DOES NOT ACTIVATE COAGULATION (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1306;
We consistently observe an inflammatory cellular phenotype and transcriptome which is, however, entirely unaffected by the presence of SARS-CoV-2. These studies support the expectation of safety of Obnitix use in COVID-ARDS.
- Ovastat (treosulfan) / Medac, Medexus, Orencia (abatacept) / BMS, Actemra IV (tocilizumab) / Roche, JW Pharma
RESULTS OF ALLOGENIC HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR RELAPSED AND REFRACTORY ACUTE LEUKEMIA: SINGLE CENTER EXPERIENCE (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1126;
These studies support the expectation of safety of Obnitix use in COVID-ARDS. Conditioning regimens: ALL
- Ovastat (treosulfan) / Medac, Medexus, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
A SUCCESSFUL OUTCOME OF DONOR-SPECIFIC ANTIBODIES MITIGATION IN HAEMATOPOIETIC STEM CELL TRANSPLANTATION (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1117;
Plasma exchange and IVIG (with antigen-positive platelets in case 2) to remove antibodies, with rituximab and ATG to deplete B and T cells, respectively proved effective in our experience. Both patients had a successful transplant outcome.
- Ovastat (treosulfan) / Medac, Medexus, Campath (alemtuzumab) / Sanofi
RENAL OUTCOME AND POSSIBLE ASSOCIATIONS IN SICKLE CELL PATIENTS WITH STEM CELL TRASPLANT IN OUR CENTER: A RETROSPECTIVE STUDY (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1109;
Renal disease contributes significantly to the morbidity and mortality of SCD. AKI generally occurs in the first three months after transplantation, what agrees with our study, and have a poor outcome with myeloablative conditioning.
- Ovastat (treosulfan) / Medac, Medexus
HEMATOPOIETIC STEM CELL TRANSPLANTATION IN PATIENTS WITH THALASSEMIA MAJOR: RETROSPECTIVE COMPARISON OF BUSULFAN TREOSULFAN CONDITIONING REGIMENS (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1082;
These two myeloablative conditioning regimens are safe and efficacious for thalassemia patients. Additionally, the introduction of new prophylaxis strategies and application of highly stringent criteria for HLA typing for donor selection are fundamental for the safety of HSCT in thalassemia.
- Ovastat (treosulfan) / Medac, Medexus
INCIDENCE AND RISK FACTORS OF GRAFT FAILURE AFTER CORD BLOOD HEMATOPOIETIC STEM CELL TRANSPLANTATION IN CHILDREN WITH MUCOPOLYSACCHARIDOSIS : A SFGM-TC RETROSPECTIVE MULTICENTER STUDY (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1074;
The use of the most compatible and richest graft possible, especially in cases of advanced age at diagnosis, would reduce the risk of GF. Monitoring the pharmacokinetics of Busulfan should also be important to improve engraftement.
- Ovastat (treosulfan) / Medac, Medexus
TREOSULFAN BASED CONDITIONING FOR HAEMATOPOIETIC STEM CELL TRANSPLANTATION (HSCT) IN PAEDIATRIC MALIGNANT DISORDERS: ON BEHALF OF THE UK PAEDIATRIC BMT GROUP (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1049;
The most common conditioning regimen was Fludarabine-treosulfan-thiotepa in 123 (85%) patients...Serotherapy was ATG (n=45, 31%), Alemtuzumab (n=50, 35%) and none (n=49, 34%).Median days to neutrophil and platelet recovery was 18 (range of 10-41) and 22 days (range of 9-81) respectively... Treosulfan based conditioning is safe in paediatric malignant disorders with low TRM and VOD rates.
- Venclexta (venetoclax) / Roche, AbbVie
SMART CONDITIONING WITH VENETOCLAX ENHANCED SEQUENTIAL FLAMSA+ALKYLATOR IN PATIENTS WITH HIGH-RISK MYELOID MALIGNANCIES (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1036;
This may be a smart way to extend the limited therapy options of patients with high-risk myeloid malignancies. To further validate these insights and enhance the idea of smart conditioning, a controlled prospective clinical trial should be the next step.
- ALLOGENEIC HCT RESOLVES INFLAMMATORY AND INFECTIOUS MANIFESTATIONS OF CHRONIC GRANULOMATOUS DISEASE IN AFFECTED FEMALE X-LINKED CARRIERS (Room 253) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1007;
Restoration of %NOB in symptomatic XL-CGD carriers by HCT may reverse inflammatory and infective manifestations and offers cure in severely affected patients. The multimorbidity of this cohort is reflected in transplant-related mortality of two out of seven patients.
- Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi
TEN-YEAR SINGLE-CENTER EXPERIENCE OF TCR??/CD19 DEPLETION FOR HEMATOPOIETIC STEM CELL TRANSPLANTATION IN INBORN ERRORS OF IMMUNITY (Room 253) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1006;
Pre-HSCT conditioning and serotherapy regimens may significantly influence transplant outcomes, whilst post-HSCT immunosuppression does not. Increased risk of GF after TCR??/CD19 depletion may require stronger myeloablation, and non-toxic agents, enhancing myeloablation effects, such as plerixafor, are of particular interest.
- HAPLOIDENTICAL ?? T CELL DEPLETED HSCT REPRESENTS A CURATIVE ALTERNATIVE TO MSD IN PATIENTS WITH TRANSFUSION DEPENDENT THALASSEMIA (Room 251) - Feb 11, 2023 - Abstract #EBMT2023EBMT_982;
All patients received an identical conditioning regimen consisting of treosulfan, thiotepa, fludarabine (FTT) and ATG-Grafalon, with the only difference in the timing of ATG (upfront in T-haplo-HSCT, prior to d0 in MSD-HSCT). Immunosuppression (IST) consisted of a combination of calcineurin inhibitors (mainly tacrolimus, and in two cases cyclosporine A) and mycophenolate mofetil...The conditioning regimen was well tolerated with no high-grade transplant related toxicity.ResultsMSDT-haplo SCTFollow-up (months)Median (range)21 (8 - 50)28 (7 - 74)Engraftment (day)Median (range)31 (20-45)18 (12-58)ChimerismMedian (range)92.2% (24.2% - 100%)100% (45.3% - 100%)Transfusion independencyMedian (range); days100 %34 (9-469)91%85 (6-298)Withdrawal IST (day)Median (day)173 (107-227)226 (112-347; 2 pts still under IST)Immunreconstitution >50 CD4+/
- Ovastat (treosulfan) / Medac, Medexus, Campath (alemtuzumab) / Sanofi
EXCELLENT GVHD-FREE, DISEASE-FREE SURVIVAL AFTER HAPLOIDENTICAL HSCT WITH PTCY FOR SICKLE CELL DISEASE (Room 251) - Feb 11, 2023 - Abstract #EBMT2023EBMT_981;
Immunosuppression (IST) consisted of a combination of calcineurin inhibitors (mainly tacrolimus, and in two cases cyclosporine A) and mycophenolate mofetil...The conditioning regimen was well tolerated with no high-grade transplant related toxicity.ResultsMSDT-haplo SCTFollow-up (months)Median (range)21 (8 - 50)28 (7 - 74)Engraftment (day)Median (range)31 (20-45)18 (12-58)ChimerismMedian (range)92.2% (24.2% - 100%)100% (45.3% - 100%)Transfusion independencyMedian (range); days100 %34 (9-469)91%85 (6-298)Withdrawal IST (day)Median (day)173 (107-227)226 (112-347; 2 pts still under IST)Immunreconstitution >50 CD4+/ Patients with severe SCD undergoing HSCT from 2014 to 2022 with unmanipulated bone marrow from parental HLA-haploidentical donors received conditioning with alemtuzumab 0,4mg/kg, fludarabine 150mg/m
- Ovastat (treosulfan) / Medac, Medexus, Jakafi (ruxolitinib) / Novartis, Incyte, Promacta (eltrombopag) / Novartis
PRE-TRANSPLANT SPLENIC RADIOTHERAPY IN PATIENTS AFFECTED BY HIGH-RISK IDIOPATHIC/SECONDARY MYELOFIBROSIS UNDERGOING ALLOGENEIC HEMATOPOIETIC STEM CELL THERAPY (HSCT) (Room 153) - Feb 11, 2023 - Abstract #EBMT2023EBMT_934;
Patients with severe SCD undergoing HSCT from 2014 to 2022 with unmanipulated bone marrow from parental HLA-haploidentical donors received conditioning with alemtuzumab 0,4mg/kg, fludarabine 150mg/m All patients received a conditioning regimen based on treosulfan (42 g/m2) and fludarabine (150 mg/m2) as per our institutional guidelines; 36% received an intensified conditioning with the addition of melphalan...Patients received PT/Cy and sirolimus as GvHD prophylaxis; MMF was added in MUD and haploidentical donors; one patient received additional ATG due to the high graft CD3+ count.Median graft CD34+ and CD3+ cell doses were respectively 6.92x10^6/Kg (1.62
- Ovastat (treosulfan) / Medac, Medexus
LONG-TERM DATA OF THE RANDOMIZED TRIAL COMPARING TBI-CONTAINING CONDITIONING REGIMEN TO A CHEMOTHERAPY-BASED PREPARATION IN CHILDREN WITH ALL. RESULTS OF THE FORUM RANDOMIZED CLINICAL TRIAL (Room 143) - Feb 11, 2023 - Abstract #EBMT2023EBMT_899;
P2/3
With a significantly longer follow-up, we confirm that the use of TBI during the conditioning regimen is associated with better outcomes in children and adolescents with ALL above the age of 4 years transplanted in CR, mainly due to a lower risk of leukemia recurrence. The number of cases of secondary malignancies, particularly in patients offered radiotherapy, underlines the importance of continuous monitoring over time to comprehensively evaluate the long-term sequels.
- Ovastat (treosulfan) / Medac, Medexus
OUTCOME OF FLUDARABINE AND TREOSULFAN CONDITIONING FOR HAEMATOPOIETIC STEM CELL TRANSPLANTATION IN 415 CHILDREN WITH NON-SCID IEI: A MULTICENTRE RETROSPECTIVE COHORT ANALYSIS (MAILLOT) - Feb 11, 2023 - Abstract #EBMT2023EBMT_845;
The number of cases of secondary malignancies, particularly in patients offered radiotherapy, underlines the importance of continuous monitoring over time to comprehensively evaluate the long-term sequels. 273 (66%) received treosulfan
- Ovastat (treosulfan) / Medac, Medexus
CLINICAL AND NEUROLOGICAL OUTCOME IN PURINE NUCLEOSIDE PHOSPHORYLASE DEFICIENT CHILDREN AFTER ALLOGENEIC HSCT - A MULTI-CENTER STUDY (MAILLOT) - Feb 11, 2023 - Abstract #EBMT2023EBMT_844;
HSCT providing sufficient myeloid DC ensures detoxification, cure of immunodeficiency and stabilization of neurological disease. Neurological outcome varied between satisfactory and severely neurologically disabled.
- Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi
ALLOGENIC STEM CELL TRANSPLANTATION WITH TCR??+/CD19+-DEPLETION IN INFANT LEUKEMIA, SINGLE CENTER EXPERIMENT (MAILLOT) - Feb 11, 2023 - Abstract #EBMT2023EBMT_840;
All major outcomes were equivalent between transplantation from unrelated, haploidentical and related donor. Low cGVHD rate ensures excellent quality of life in the survivors.
- Ovastat (treosulfan) / Medac, Medexus, Grafalon (rabbit anti-T-lymphocyte globulin) / Mundipharma, NeoPharm
LOW TOXICITY WITH MYELOABLATIVE CHEMO-BASED CONDITIONING FOR CHILDREN WITH ALL BELOW 4 YEARS OF AGE. RESULTS FROM THE PROSPECTIVE MULTINATIONAL FORUM-TRIAL (MAILLOT) - Feb 11, 2023 - Abstract #EBMT2023EBMT_834;
P2/3
Within the FORUM-trial, infants and young children receiving HSCT after conditioning with either BU- or TREO containing regimens have a lower OS and EFS as compared to children above the age of 4 yrs due to a higher CIR. Because of decreased NRM, these results represent an improvement as compared to previously reported series; however, KMT2A-rearrangement continues to be an obstacle to successful HSCT.
- Ovastat (treosulfan) / Medac, Medexus, Eloctate (efraloctocog alfa) / Sanofi, SOBI
HSCT in a newborn suffering from SCID and severe haemophilia A (#125) (foyer) - Feb 8, 2023 - Abstract #GTH2023GTH_384;
The first substitution of elevated half-life factor VIII product (Efmoroctocog alfa) was required during implantation of the central venous catheter...After HSCT, the inhibitor risk presumably remains decreased due to the fact that the healthy donor cells were exposed to normal factor VIII levels. However, this is speculative and further studies are needed to investigate haemophilia patients undergoing HSCT and haemophilia patients suffering from immunodeficiencies.
- oNKord (GCT NK cells) / Glycostem, Medac, Korea Kolmar
Universal prospects of cryopreserved umbilical cord blood CD34+ progenitor cell-derived NK cells: Clinical and preclinical evaluation of non-engineered and genetically engineered candidates (Hall Bordeaux) - Jan 31, 2023 - Abstract #ESMOTAT2023ESMO_TAT_137;
Furthermore, approaches to genetically equip GTA002 with chimeric antigen receptors to generate viveNK TM cells, recently showed efficient preclinical antigen-specific targeting of HER2 + and CD19 + tumors, while preserving innate NK cell cytotoxicity. Conclusions Overall, the preclinical innate performance and ADCC of cryopreserved
- || Jivadco (trastuzumab duocarmazine) / Byondis, Medac
Trial completion date, Trial primary completion date, Metastases: SYD985 in Patients With HER2-expressing Recurrent, Advanced or Metastatic Endometrial Carcinoma (clinicaltrials.gov) - Jan 26, 2023
P2, N=60, Active, not recruiting, Sponsor: Byondis B.V.
Conclusions Overall, the preclinical innate performance and ADCC of cryopreserved Trial completion date: Dec 2022 --> May 2023 | Trial primary completion date: Dec 2022 --> Apr 2023
- || Jivadco (trastuzumab duocarmazine) / Byondis, Medac
Trial completion date, Trial primary completion date, Combination therapy, Metastases: Phase I Study of SYD985 With Niraparib in Patients With Solid Tumors (clinicaltrials.gov) - Jan 25, 2023
P1, N=120, Active, not recruiting, Sponsor: Byondis B.V.
Trial completion date: Dec 2022 --> May 2023 | Trial primary completion date: Dec 2022 --> Apr 2023 Trial completion date: Jan 2023 --> Dec 2023 | Trial primary completion date: Dec 2022 --> Jun 2023
- | Blincyto (blinatumomab) / Astellas, Amgen
Trial completion date: AALL1331: Blinatumomab in Treating Younger Patients With Relapsed B-cell Acute Lymphoblastic Leukemia (clinicaltrials.gov) - Jan 25, 2023
P3, N=669, Active, not recruiting, Sponsor: National Cancer Institute (NCI)
Trial completion date: Jan 2023 --> Dec 2023 | Trial primary completion date: Dec 2022 --> Jun 2023 Trial completion date: Dec 2030 --> Jun 2023
- Donor-derived CAR-T Cells Co-infusion with T-depleted HSCT (Poster Area) - Jan 24, 2023 - Abstract #EHAEBMTCART2023EHA_EBMT_CART_55;
P1/2
Allogeneic haploidentical donor-derived CAR-T cells can be safely co-infused with the αβ-T cell-depleted grafts, with minimal CAR-T-related toxicity, without compromise of engraftment and GVHD control. CAR-T cells expand and persist as expected.