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46 Trials |  |
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- | lestaurtinib (CEP-701) / Teva, Kyowa Kirin
Enrollment closed: AALL0631: Combination Chemotherapy With or Without Lestaurtinib in Treating Younger Patients With Newly Diagnosed Acute Lymphoblastic Leukemia (clinicaltrials.gov) - Mar 24, 2020
P3, N=218, Active, not recruiting, Sponsor: Children's Oncology Group
Completed --> Active, not recruiting
- || Orencia (abatacept) / BMS, Actemra IV (tocilizumab) / Roche, JW Pharma
Clinical, Journal: αβ T cell-depleted haploidentical hematopoietic stem cell transplantation without anti-thymocyte globulin in children with chemorefractory acute myeloid leukemia. (Pubmed Central) - Mar 22, 2020
Antithymocyte globulin was substituted with tocilizumab in all patients and also with abatacept in 10 patients...At 2 years the transplant-related mortality was 9%, relapse incidence was 42%, event-free survival was 49%, and overall survival was 53%. Our data suggest that αβ T cell-depleted haploidentical HSCT provides a reasonable chance of long-term survival in a cohort of children with chemorefractory acute myeloid leukemia and creates a solid basis for further improvement.
- | trastuzumab duocarmazine (SYD985) / Synthon
Journal: The Second Generation Antibody-Drug Conjugate SYD985 Overcomes Resistances to T-DM1. (Pubmed Central) - Mar 19, 2020
Characterization of these models showed that previously described mechanisms-HER2 downmodulation, impairment of lysosomal function and upregulation of drug efflux pumps-account for the resistances observed, arguing that mechanisms of resistance to T-DM1 are limited, and most of them have already been described. Importantly, SYD985 was effective in these models, showing that the resistance to first generation ADCs can be overcome with an improved design.
- | Ovastat (treosulfan) / Medac
Preclinical, Journal: Kinetics of In Vitro Guanine- N7-Alkylation in Calf Thymus DNA by (2 S,3 S)-1,2-Epoxybutane-3,4-Diol 4-Methanesulfonate and (2 S,3 S)-1,2:3,4-Diepoxybutane: Revision of the Mechanism of DNA Cross-linking by the Prodrug Treosulfan. (Pubmed Central) - Mar 18, 2020
In conclusion, EBDM can produce DNA-DNA lesions independently of DEB, and likely plays a greater role in DNA cross-linking after in vivo administration of treosulfan than DEB. These findings compel revision of the previously proposed mechanism of the pharmacological action of treosulfan and contribute to better understanding of the importance of EBDM for biological effects.
- || Metoject (methotrexate autopen) / Medac
Qu’en est-il des autres traitements? Metoject, etc. (Twitter) - Mar 17, 2020
- | thioguanine / Generic mfg.
Trial primary completion date: Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome (clinicaltrials.gov) - Mar 13, 2020
P3, N=256, Recruiting, Sponsor: Children's Oncology Group
These findings compel revision of the previously proposed mechanism of the pharmacological action of treosulfan and contribute to better understanding of the importance of EBDM for biological effects. Trial primary completion date: Mar 2022 --> Mar 2023
- || Ovastat (treosulfan) / Medac
Biomarker, Clinical, Journal: A simplified method for detection of N-terminal valine adducts in patients receiving treosulfan. (Pubmed Central) - Mar 8, 2020
The mean ± SD amounts of adduct formed were 245.3 ± 89.6 and 210 ± 78.5 pmol/g globin for pyr-Val and THB-Val, respectively. Importantly, these results show that this direct injection method can quantitate both background and treosulfan-induced pyr-Val and THB-Val N-terminal valine globin adducts in humans.
- | Ovastat (treosulfan) / Medac, Medexus
Trial initiation date: Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Total-Body Irradiation for the Treatment of Hematological Malignancies (clinicaltrials.gov) - Mar 4, 2020
P2, N=60, Not yet recruiting, Sponsor: Fred Hutchinson Cancer Research Center
Importantly, these results show that this direct injection method can quantitate both background and treosulfan-induced pyr-Val and THB-Val N-terminal valine globin adducts in humans. Initiation date: Mar 2020 --> Jul 2020
- | Ovastat (treosulfan) / Medac, fludarabine / Generic mfg.
Journal: Mismatched related versus matched unrelated donors in TCRαβ/CD19-depleted HSCT for primary immunodeficiencies. (Pubmed Central) - Mar 3, 2020
In our experience, there was no difference in the outcomes of HSCT performed from MUD and MMRD. Hence, given the immediate availability of donors, in the absence of HLA-identical siblings HSCT with TCRαβ+/CD19+ graft depletion from MMRDs can be considered as the first choice in patients with PID.
- | Xospata (gilteritinib) / Astellas, Vyxeos (cytarabine/daunorubicin liposomal formulation) / Jazz, Nippon Shinyaku, Mylotarg (gemtuzumab ozogamicin) / UCB, PDL, Pfizer
New P3 trial: A Study to Compare Standard Chemotherapy to Therapy With CPX-351 and/or Gilteritinib for Patients With Newly Diagnosed AML With or Without FLT3 Mutations (clinicaltrials.gov) - Mar 3, 2020
P3, N=1400, Not yet recruiting, Sponsor: Children's Oncology Group
- ||| Enhertu (fam-trastuzumab deruxtecan-nxki) / Daiichi Sankyo, AstraZeneca, trastuzumab duocarmazine (SYD985) / Synthon
Agree, there’s a lot of potential for an agnostic activity in HER2 expressing tumors. We already have promising data of DS8201 beyond BC, in gastric cancer and CRC. The ADC SYD985 has also been tested in various HER2-expressing tumors, with activity seen in several histologies (Twitter) - Feb 15, 2020
- || Ovastat (treosulfan) / Medac, fludarabine / Generic mfg., methotrexate / Generic mfg.
Clinical, Journal: Risk Factors and Implications of Oral Mucositis in Recipients of Allogeneic Hematopoietic Stem Cell Transplantation. (Pubmed Central) - Feb 14, 2020
Oral mucositis remains a leading SCT complication. Treosulfan-based conditioning has low mucosal toxicity and is appealing given previous reports on its high efficacy.
- | Trial completion, Trial completion date, Metastases: Low Dose Metronomic Poly-chemotherapy for Metastatic CRC (clinicaltrials.gov) - Feb 12, 2020
P2, N=45, Completed, Sponsor: HaEmek Medical Center, Israel
Treosulfan-based conditioning has low mucosal toxicity and is appealing given previous reports on its high efficacy. Active, not recruiting --> Completed | Trial completion date: Jan 2019 --> Dec 2019
- | Biomarker, Enrollment change, PARP Biomarker, PD(L)-1 Biomarker: I-SPY 2: I-SPY TRIAL: Neoadjuvant and Personalized Adaptive Novel Agents to Treat Breast Cancer (clinicaltrials.gov) - Feb 10, 2020
P2, N=4000, Recruiting, Sponsor: QuantumLeap Healthcare Collaborative
Active, not recruiting --> Completed | Trial completion date: Jan 2019 --> Dec 2019 N=1920 --> 4000
- sirolimus / Generic mfg.
[VIRTUAL] INTRA-BONE CORD-BLOOD TRANSPLANT OF UNWASHED UNITS WITH ATG-FREE AND SIROLIMUS-BASED GVHD PROPHYLAXIS: FAST IMMUNE-RECONSTITUTION AND LONG-TERM DISEASE CONTROL IN 22 PATIENTS WITH HIGH-RISK DISEASES (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2378;
Intra-bone infusion of diluted unwashed cord-blood units reduces cell loss and is safe and easy to perform. Our data confirm that calcineurin-free, ATG-free GvHD prophylaxis is associated with full hematopoietic engraftment, good profiles of immune-reconstitution and low rates of GvHD; considering the very high-risk population, our data suggest interesting long-term disease control, deserving further investigation in early phase disease.
- Ovastat (treosulfan) / Medac
[VIRTUAL] TREOSULFAN EXPOSURE IN PEDIATRIC HEMATOPOIETIC STEM CELL TRANSPLANTATION IS ASSOCIATED WITH EARLY TOXICITY BUT NOT WITH EVENT-FREE SURVIVAL (Auditorium 1) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2246;
We demonstrate a relationship between treosulfan exposure and mucosal and skin toxicity, which is most pronounced in young children. Treosulfan-based conditioning is safe and effective, particularly in infants.
- tacrolimus / Generic mfg., Enbrel (etanercept) / Pfizer, Amgen, cyclosporine / Generic mfg.
[VIRTUAL] USE OF SUBLINGUAL TACROLIMUS IN PEDIATRIC HEMATOPOIETIC STEM CELL TRANSPLANT RECIPIENTS WITH HISTORY OF ANAPHYLAXIS TO INTRAVENOUS CYCLOSPORINE (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2226;
Sublingual tacrolimus should be considered as safe and reliable alternative to IV tacrolimus in cases where oral form cannot be used. It can also become an option to oral or parenteral form of cyclosporine.
- tacrolimus / Generic mfg.
[VIRTUAL] HAPLOIDENTICAL T-CELL DEPLETED HSCT REPRESENTS A SAFE THERAPEUTIC ALTERNATIVE TO MSD TRANSPLANTATION IN PATIENTS WITH TRANSFUSION-DEPENDENT ß-THALASSEMIA MAJOR (Auditorium 4) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2218;
These preliminary data of a small patient cohort with TDT add confirmative evidence that T-haplo-HSCT represents a safe alternative for TDT patients without a MSD. Treosulfan demonstrated to be an excellent alternative to busulfan, with no case of veno-occlusive disease in this high-risk patient population.
- [VIRTUAL] THE ROLE OF HLA MATCHING IN UNRELATED DONOR HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR SICKLE CELL DISEASE IN EUROPE (Auditorium 4) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2217;
Using a 10/10 HLA-matched unrelated donor yields better OS and GFRS in comparison to mismatched donors. Also, the use of FLU-THIO-TREO as conditioning regimen is associated with better survival.
- cyclophosphamide intravenous / Generic mfg.
[VIRTUAL] THE EXPERIENCE OF HEMATOPOIETIC STEM CELL TRANSPLANTATION IN RARE HEMATOLOGIC DISEASE (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2209;
HSCT is the only curative treatment for some life-threatening rare diseases. Clinical Trial Registry: No
- Flagyl (metronidazole) / SLA, tacrolimus / Generic mfg.
[VIRTUAL] PNEUMATOSIS INTESTINALIS AS A RARE POST-TRANSPLANT COMPLICATION IN PEDIATRIC PATIENTS (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2207;
Both cases confirm the previously described association of PI with GvHD and intense immunosuppression. While conservative management was successful in one asymptomatic patient, an emergency diagnostic laparotomy could not be avoided in the other patient due to acute abdominal pain.
- EVOLUTION IN HAEMATOPOIETIC CELL TRANSPLANT (HCT) FOR PRIMARY IMMUNODEFICIENCY DISORDERS (PID) IN A SINGLE PAEDIATRIC CENTER (Poster Area (Exhibition Area)) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2204;
Advances in graft manipulation allows access to cure with excellent results. Use of target therapy /adoptive cell therapy can be used as a bridge to HCT.
- Venclexta (venetoclax) / Roche, AbbVie, azacitidine / Generic mfg., decitabine / Generic mfg.
[VIRTUAL] VENETOCLAX/DECITABINE FOR PEDIATRIC MDR-AML RELAPSED AFTER TWO ALLO HSCTS (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2197;
This represents a promising therapeutic approach for otherwise untreatable relapses after HSCT, but questions remain about the long-term durability of remission. Future studies should also aim at identifying patients who will profit the most from this therapy.
- tacrolimus / Generic mfg., sirolimus / Generic mfg.
[VIRTUAL] OUTCOME OF ALLOGENEIC STEM CELL TRANSPLANTATION (ALLOHSCT) WITH DIFFERENT INTENSITY CONDITIONING REGIMENS IN INFANT AML (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2196;
RIC efficacy is equal to MAC in infant AML on the basis of OS, RFS, GRFS, CRI and TRM and especially has to be considered in CR1. Potentially RIC may reduce long-term severe side effects of alloHSCT correlated with toxic effect of conditioning regimen intensity.
- Ovastat (treosulfan) / Medac, melphalan / Generic mfg., busulfan / Generic mfg.
[VIRTUAL] CONDITIONING FOR RETRANSPLANTATION FROM THE SAME DONOR AFTER PARTIAL AUTOLOGOUS RECONSTITUTION IN PATIENTS WITH PID: IMMUNOSUPPRESSION IS NOT REQUIRED (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2192;
In summary these data demonstrate that a selective myelosuppressive conditioning regimen for retransplantation from the same donor after autologous myeloid reconsititution is a feasible approach to achieve stable myeloid engraftment with low toxicity, reduced treatment related mortality or infectious complications. In summary these data demonstrate that a selective myelosuppressive conditioning regimen for retransplantation from the same donor after autologous myeloid reconsititution is a feasible approach to achieve stable myeloid engraftment with low toxicity or treatment related mortality.
- [VIRTUAL] AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR PEDIATRIC HIGH RISK EWING SARCOMA, A SINGLE CENTER EXPERIENCE (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2191;
Patients who experience local relapse following HSCT could be salvaged as opposed to patients who relapsed in multiple sites. The efficacy of HSCT in pediatric patients with ES should be evaluated in larger randomized control studies.
- Ovastat (treosulfan) / Medac, fludarabine / Generic mfg., thiotepa / Generic mfg.
[VIRTUAL] IS ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION A GOOD INDICATION FOR PATIENTS WITH SIFD SYNDROME? (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2189;
Allogeneic HSCT can improve /resolve hematological and immunological aspects in severely affected patients. However, successful HSCT could not improve the metabolic/neurological aspects of this enzyme deficiency.
- Orencia (abatacept) / BMS, Vistide (cidofovir) / Gilead, sirolimus / Generic mfg.
[VIRTUAL] SUCCESSFUL HSCT IN IMMUNE DISREGOLATORY DISEASES (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2183;
The increasing control of post-transplant complications with the reduction of TRM also allows us to submit to HCST also pathologies that do not have an explicit indication. The outcome is one of better quality of life with the resolution of disabling symptoms.
- [VIRTUAL] BOOST WITH CD34+ CELLS FOR POOR GRAFT FUNCTION TREATMENT AFTER HSCT IN PAEDIATRIC PATIENTS. A MULTICENTER STUDY (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2178;
However, this approach seems to be able to rescue only approximately one half of patients developing PGF in the long term. Clinical Trial Registry: NA
- Ovastat (treosulfan) / Medac, melphalan / Generic mfg., busulfan / Generic mfg.
[VIRTUAL] IMPROVED SURVIVAL AFTER MIBG THERAPY IN COMBINATION WITH MEGACHEMOTHERAPY THAN AFTER STAND-ALONE THERAPY IN CHILDREN WITH NEUROBLASTOMA - POLISH NATION-WIDE RETROSPECTIVE STUDY (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2164;
The VOD incidence did not seem to be affected by the MIBG megatherapy. The thyroid gland failure showed increasing incidence trend over the follow-up time.
- [VIRTUAL] DETECTION OF RECIPIENT-SPECIFIC α/β-T-CELLS DURING THE PRE-ENGRAFTMENT PHASE DOES NOT PREDICT GRAFT REJECTION IN PATIENTS AFTER HAPLOSCT: PRELIMINARY RESULTS OF SINGLE CENTRE PROSPECTIVE TRIAL (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2163;
Flow-cytometry based analysis of hematopietic chimerism in different cell populations is reliable and characterised by moderate to substantial agreement with standard STR-PCR. Despite limited sample size, the early results indicate that detection of up to 100% autologous α/β-T-cells in pre-engraftment phase does not predict graft rejection.
- Ovastat (treosulfan) / Medac, busulfan / Generic mfg.
[VIRTUAL] HAEMATOPOIETIC STEM CELL TRANSPLANTATION IN PATIENTS WITH HURLER SYNDROME: SINGLE CENTER EXPERIENCE (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2158;
Pretransplantation accumulation of acid glycosaminoglycans leads to the development of organ disorders, especially liver and lungs. The choice of donor and optimal conditioning regimen are a factors affecting the effectiveness of treatment.
- Campath (alemtuzumab) / Sanofi, tacrolimus / Generic mfg., cyclophosphamide intravenous / Generic mfg.
[VIRTUAL] COMBINED TCR ALPHA BETA AND CD45RA DEPLETED HAPLOIDENTICAL STEM CELL TRANSPLANT IN CHILDREN WITH BENIGN HAEMATOLOGICAL CONDITIONS, SINGLE CENTRE EXPERIENCE (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2149;
TCR alpha beta and CD45RA depleted haploidentical transplant provides fast engraftment, minimal GVHD risk, and good early immune reconstitution. Mixed chimerism was common and secondary graft failure developed in 2 children both managed successfully with DLI and second transplant with 100% survival in this cohort of children.
- cyclophosphamide intravenous / Generic mfg.
[VIRTUAL] PHARMACOKINETICS OF ACTIVE ATG IMPACTS T-CELL RECOVERY AFTER TCRαβ/CD19-DEPLETED HSCT IN PAEDIATRIC PATIENTS (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2145;
Although this did not affect the clinical outcome in our study, we believe that ATG clearance and, therefore, the timing of T-cell recovery may be critically important especially for patients treated with a mismatched donor. Future studies, with clinical cohorts of non-malignant hematologic diseases and the TCRαβ/CD19-depleted HSCT setting, are necessary to increase our knowledge on ATG pharmacokinetics and dynamics, eventually leading to further optimization of ATG treatment.
- Ovastat (treosulfan) / Medac, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
[VIRTUAL] HEMATOPOIETIC STEM CELL TRANSPLANTATION (HSCT) IN REFRACTORY CYTOPENIA OF CHILDHOOD (RCC): IMPROVED ENGRAFTMENT FOLLOWING A TREOSULFAN BASED CONDITIONING REGIMEN (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2144;
The reduced toxicity conditioning regimen consisting of treosulfan and fludarabine resulted in an excellent outcome with sustained engraftment and low incidence of GvHD in this cohort of patients with RCC. Long term follow up is needed to investigate the incidence of late effects, including the risk of infertility.
- Ovastat (treosulfan) / Medac, fludarabine IV / Generic mfg.
[VIRTUAL] HAPLOIDENTICAL DONOR OR UNRELATED DONOR FOR INFANTS WITH SEVERE COMBINED IMMUNODEFICIENCY? (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2141;
Immune reconstitution kinetics and donor chimerism after HID were comparable with MFD and MUD. In our center, haploidentical donor is preferred than MUD in patients without MFD.
- Orencia (abatacept) / BMS, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
[VIRTUAL] RESULTS OF HEMATOPOIETIC STEM CELLS TRANSPLANTATION FROM MATCHED RELATED DONOR WITH TCRαβ+/CD19+-DEPLETION AND A FIXED DOSE MEMORY T CELL (CD45RA-DEPLETED) ADD-BACK (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2072;
Patients with primary immune deficiency received fludarabine, ATG and either busulfan (n=3) or treosulfan (n=3)...A patient with thalassemia (n-1) - treosulfan, thiotepa, fludarabine, ATG...As GVHD prophylaxis we used bortezomib (n-16), rituximab (n-19) and abatacept (n-19)... We confirm that the depletion of αβ T cells from related graft in combination with intensive conditioning regimen and additional infusion 1x106/kg CD45RO T cells on day 0 provides is associated with high rate of engraftment, very low risk of GVHD, absent TRM, and promising long-term survival in a cohord of children with malignant and non-malignant HSCT indications.
- cyclophosphamide oral / Generic mfg.
[VIRTUAL] SUCCESFUL HLA-MISMATCHED HSCT IN PAEDIATRIC PATIENTS WITH HEMOGLOBINOPATHIES USING ATG SEROTHERAPY AND POSTTRANSPLANT CYCLOPHOSPHAMIDE (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2061;
Delayed neutrophil and early T-cell recovery in the PT-CY group did not negatively impact outcome. Therefore, in the absence of a matched donor, a mismatched donor using the platform of treosulfan-based myeloablative conditioning, ATG and PT-CY may be considered an equally safe and effective treatment option.
- [VIRTUAL] THE IMPACT OF CONDITIONING IN CHILDREN AND ADOLESCENTS WITH ALL: A PROSPECTIVE RANDOMIZED MULTICENTER-STUDY “FORUM” ON BEHALF OF AIEOP-BFM-ALL-SG, IBFM-SG, INTREALL-SG AND EBMT-PD-WP (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2051;
P2/3
There is no significant difference in 2-year TRM and incidence of acute and chronic GVHD. TBI/etoposide should be recommended as standard conditioning regimen with prospective monitoring of late complications, endocrine functions and incidence of secondary malignancies.
- Ovastat (treosulfan) / Medac, melphalan / Generic mfg.
[VIRTUAL] FERTILITY IN ADULT FEMALE ALLOGENEIC STEM CELL RECIPIENTS (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_2040;
Post-transplant pregnancy is possible even after ovotoxic TBI. HCT at younger ages, non-malignant diseases and conditioning with Treosulfan could be shown to have fertility preserving potential.
- Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
[VIRTUAL] ANTI-T-LYMPHOCYTE GLOBULIN (ATLG) AND RITUXIMAB FOR IMMUNOMODULATION OF GRAFT-VERSUS-HOST DISEASE AND GRAFT FAILURE IN PATIENTS WITH NON-MALIGNANT DISORDERS: RESULTS OF A MULTICENTRE, RANDOMIZED, OPEN-LABEL STUDY (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_1988;
P2
In MUD-HSCT recipients, the administration of a fixed dose of pre-transplant rituximab does not affect the risk of aGvHD and transplant-related mortality, while it significantly reduces the incidence of EBV-viremia episodes, without affecting B-cell recovery. Clinical Trial Registry: ClinicalTrials.gov Identifier: NCT01810926 https://clinicaltrials.gov/ct2/show/NCT01810926 EudraCT Number: 2011-004730-34 https://www.clinicaltrialsregister.eu/ctr-search/trial/2011-004730-34/IT
- Orencia (abatacept) / BMS, Actemra IV (tocilizumab) / Roche, JW Pharma, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
[VIRTUAL] TCRαβ+/CD19+-DEPLETION IN HEMATOPOIETIC STEM CELLS TRANSPLANTATION FROM MATCHED UNRELATED AND HAPLOIDENTICAL DONORS IN CHILDREN WITH HIGH-RISK ACUTE MYELOBLASTIC LEUKEMIA IN FIRST COMPLETE REMISSION (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_1974;
This analysis indicates that αβ T-depleted HSCT from haploidentical donors provides a high chance of long-term survival in a cohort of children with high-risk AML in CR1 and is associated with a low burden of GVHD and non-relapse mortality. We suggest that due to easy logistics donor choice for children with high-risk AML in CR1 should be reconsidered in favor of haploidentical donors.
- Ovastat (treosulfan) / Medac, busulfan / Generic mfg.
[VIRTUAL] EBMT SURVEY ON 40 YEARS OF HIGH-DOSE CHEMOTHERAPY (HDT) AND STEM CELL TRANSPLANTATION (SCT) IN EWING TUMOURS (ET). A REPORT FROM THE PAEDIATRIC WORKING PARTY (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_1972;
P3
This EBMT Ewing data set holds important information for decision making processes and suggests exploring in more depth the results and roles of Busulfan versus Treosulfan in front line trials. Clinical Trial Registry: NCT00987636
- Ovastat (treosulfan) / Medac, busulfan / Generic mfg.
[VIRTUAL] EBMT LESSONS FROM 40 YEARS OF HIGH DOSE CHEMOTHERAPY (HDT) AND STEM CELL TRANSPLANTATION (SCT) IN NEUROBLASTOMA: A REPORT FROM THE PAEDIATRIC WORKING PARTY (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_1971;
Others like double transplants need cautious interpretation as mostly applied after multiple lines of conventional treatments or at relapse in the EBMT data base. Hence more recently reported superiority in the front line setting is not visible yet.
- Orencia (abatacept) / BMS, Actemra IV (tocilizumab) / Roche, JW Pharma
[VIRTUAL] LOW-DOSE MEMORY DONOR LYMPHOCYTE INFUSION AFTER αβ T CELL-DEPLETED HSCT AMONG CHILDREN WITH HIGH-RISK LEUKEMIA: RESULTS OF A PROSPECTIVE RANDOMIZED SINGLE-CENTER TRIAL (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_1965;
P2/3
Low-dose CD45RA-depleted DLI commenced at day of grafting were safe but insufficient to prevent CMV viremia. Overall αβ T cell depleted HSCT among children with high-risk leukemia was associated with very low non-relapse mortality, which we ascribe to overall refinement of the regimen, specifically to replacement of polyclonal lymphodepleting serotherapy with targeted immunomodulation Clinical Trial Registry: NCT02942173
- Ovastat (treosulfan) / Medac
[VIRTUAL] FATHERHOOD AFTER A MYELOABLATIVE TREOSULFAN BASED ALLOGENEIC STEM CELL TRANSPLANTATION: ANALYSIS ON 453 PATIENTS (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_1756;
An adequate patient's counseling and the pursuit of strategies to preserve fertility after transplantation are essential. Treosulfan seems to be associated with a lower gonadal damage: further longitudinal cohort studies are essential for improving the care and assistance of our patients.
- Ovastat (treosulfan) / Medac, cyclophosphamide intravenous / Generic mfg.
[VIRTUAL] GONADOTOXICITY AFTER TREOSULFAN-BASED MYELOABLATIVE CONDITIONING REGIMEN FOR ALLOGENEIC HSCT IN GIRLS WITH LEUKEMIA (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_1754;
Treosulfan-based conditioning allows to preserve gonadal function in about 70% of girls after allo-HSCT. Further studies are needed to evaluate gonadal function recovery and its persistence in allo-HSCT with treosulfan-based conditioning in larger groups with longer follow-up.
- tacrolimus / Generic mfg., sirolimus / Generic mfg., cyclophosphamide intravenous / Generic mfg.
[VIRTUAL] WERNICKE'S ENCEPHALOPATHY IN A CHILD WITH B LYMPHOBLASTIC LYMPHOMA AFTER HAPLO-HSCT (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_1731;
Children after HSCT and with severe oncological diseases are at major risk of WE. Early clinical signs of WE may be not specific and evident.
- Ovastat (treosulfan) / Medac
[VIRTUAL] TREOSULFAN-INDUCED MYALGIA IN PEDIATRIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_1708;
A majority of patients required opiates to manage the pain. Although myalgia might be classified as a 'minor' side effect, it is a frequent cause of comorbidity in the early post-HSCT period.
- [VIRTUAL] PERSISTENT MIXED CHIMERISM WITHOUT RELAPSE IN CHILDREN AFTER HSCT FOR HEMATOLOGIC MALIGNANCY (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_1627;
Further study is needed to determine what factors can predict which patients with mixed chimerism will remain in remission in order to avoid the morbidity associated with DLI. Notably, the two patients who had distinction of their cells had mixed chimerism of their myeloid compartment, which may have allowed them to maintain suppression of their leukemia with a combination of NK and T lymphocytes.