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120 Diseases | | 9 Products |
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- 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) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4585;
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.
- 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) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4584;
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.
- 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) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4583;
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) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4519;
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.
- Ovastat (treosulfan) / Medac
[VIRTUAL] TREOSULFAN-INDUCED MYALGIA IN PEDIATRIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4518;
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.
- Ovastat (treosulfan) / Medac
[VIRTUAL] TREOSULFAN-INDUCED MYALGIA IN PEDIATRIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4517;
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) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4312;
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.
- [VIRTUAL] PERSISTENT MIXED CHIMERISM WITHOUT RELAPSE IN CHILDREN AFTER HSCT FOR HEMATOLOGIC MALIGNANCY (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4311;
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.
- [VIRTUAL] PERSISTENT MIXED CHIMERISM WITHOUT RELAPSE IN CHILDREN AFTER HSCT FOR HEMATOLOGIC MALIGNANCY (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4310;
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.
- busulfan / Generic mfg.
[VIRTUAL] PATTERN OF RECONSTITUTION OF RECENT THYMIC EMIGRANTS AFTER REDUCED INTENSITY CONDITIONING BASED ON TARGETED BUSULFAN IN PAEDIATRIC RECIPIENTS OF ALLOGENEIC HAEMATOPOETIC STEM CELL TRANSPLANTATION (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4225;
The cohort assessed here is exceptional with respect to the homogeneity of the RIC-regimen administered including the accuracy of the measurement of its intensity. This allowed us to better correlate conditioning intensity, occurrence of GvHD and re-emergence of thymic function after adjustment for other important factors.
- busulfan / Generic mfg.
[VIRTUAL] PATTERN OF RECONSTITUTION OF RECENT THYMIC EMIGRANTS AFTER REDUCED INTENSITY CONDITIONING BASED ON TARGETED BUSULFAN IN PAEDIATRIC RECIPIENTS OF ALLOGENEIC HAEMATOPOETIC STEM CELL TRANSPLANTATION (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4224;
The cohort assessed here is exceptional with respect to the homogeneity of the RIC-regimen administered including the accuracy of the measurement of its intensity. This allowed us to better correlate conditioning intensity, occurrence of GvHD and re-emergence of thymic function after adjustment for other important factors.
- busulfan / Generic mfg.
[VIRTUAL] PATTERN OF RECONSTITUTION OF RECENT THYMIC EMIGRANTS AFTER REDUCED INTENSITY CONDITIONING BASED ON TARGETED BUSULFAN IN PAEDIATRIC RECIPIENTS OF ALLOGENEIC HAEMATOPOETIC STEM CELL TRANSPLANTATION (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4223;
The cohort assessed here is exceptional with respect to the homogeneity of the RIC-regimen administered including the accuracy of the measurement of its intensity. This allowed us to better correlate conditioning intensity, occurrence of GvHD and re-emergence of thymic function after adjustment for other important factors.
- Orencia (abatacept) / BMS, Actemra IV (tocilizumab) / Roche, JW Pharma, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
[VIRTUAL] FLOW-BASED PRE-TRANSPLANT MRD DETECTION STRONGLY IMPACTS ON THE OUTCOME OF HAPLOIDENTICAL TRANSPLANTATION WITH AB T CELL DEPLETION IN PEDIATRIC ACUTE LYMPHOBLASTIC LEUKEMIA (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4201;
TBI regimen play a significant role only in MRD positive group in this cohort of patients. We expect that further improvement of the outcome can be achieved based on the combination of current safe haplo HSCT platform and novel targeted immunotherapy approaches.
- Orencia (abatacept) / BMS, Actemra IV (tocilizumab) / Roche, JW Pharma, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
[VIRTUAL] FLOW-BASED PRE-TRANSPLANT MRD DETECTION STRONGLY IMPACTS ON THE OUTCOME OF HAPLOIDENTICAL TRANSPLANTATION WITH AB T CELL DEPLETION IN PEDIATRIC ACUTE LYMPHOBLASTIC LEUKEMIA (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4200;
TBI regimen play a significant role only in MRD positive group in this cohort of patients. We expect that further improvement of the outcome can be achieved based on the combination of current safe haplo HSCT platform and novel targeted immunotherapy approaches.
- Orencia (abatacept) / BMS, Actemra IV (tocilizumab) / Roche, JW Pharma, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
[VIRTUAL] FLOW-BASED PRE-TRANSPLANT MRD DETECTION STRONGLY IMPACTS ON THE OUTCOME OF HAPLOIDENTICAL TRANSPLANTATION WITH AB T CELL DEPLETION IN PEDIATRIC ACUTE LYMPHOBLASTIC LEUKEMIA (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4199;
TBI regimen play a significant role only in MRD positive group in this cohort of patients. We expect that further improvement of the outcome can be achieved based on the combination of current safe haplo HSCT platform and novel targeted immunotherapy approaches.
- Ovastat (treosulfan) / Medac, busulfan / Generic mfg.
[VIRTUAL] HEMATOPOETIC STEM CELL TRANSPLANTATION IN CD40 LIGAND DEFICIENCY (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3802;
The conditioning regimens were predominantly based on myeloablative protocols that consisted of treosulfan or busulfan with weight-based dosages, except for two twin cases wherein a non-myeloablative regimen was used in their first transplantation. Our data show that HSCT for patients with CD40 ligand deficiency is a potentially effective treatment for long-term disease control.
- Ovastat (treosulfan) / Medac, busulfan / Generic mfg.
[VIRTUAL] HEMATOPOETIC STEM CELL TRANSPLANTATION IN CD40 LIGAND DEFICIENCY (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3801;
The conditioning regimens were predominantly based on myeloablative protocols that consisted of treosulfan or busulfan with weight-based dosages, except for two twin cases wherein a non-myeloablative regimen was used in their first transplantation. Our data show that HSCT for patients with CD40 ligand deficiency is a potentially effective treatment for long-term disease control.
- Ovastat (treosulfan) / Medac, busulfan / Generic mfg.
[VIRTUAL] HEMATOPOETIC STEM CELL TRANSPLANTATION IN CD40 LIGAND DEFICIENCY (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3800;
The conditioning regimens were predominantly based on myeloablative protocols that consisted of treosulfan or busulfan with weight-based dosages, except for two twin cases wherein a non-myeloablative regimen was used in their first transplantation. Our data show that HSCT for patients with CD40 ligand deficiency is a potentially effective treatment for long-term disease control.
- Campath (alemtuzumab) / Sanofi
[VIRTUAL] HAEMATOPOIETIC STEM CELL TRANSPLANTATION FOR X-LINKED AGAMMAGLOBULINAEMIA COMPLICATED BY CHRONIC NOROVIRUS ENTEROPATHY (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3799;
To our knowledge, this is the first HSCT for XLA in developed healthcare systems solely to cure XLA or its direct complications. Patients have previously been cured of XLA through HSCT, but only when HSCT was offered for a haematological co-morbidity (e.g. Sickle Cell Disease, Leukaemia).
- Campath (alemtuzumab) / Sanofi
[VIRTUAL] HAEMATOPOIETIC STEM CELL TRANSPLANTATION FOR X-LINKED AGAMMAGLOBULINAEMIA COMPLICATED BY CHRONIC NOROVIRUS ENTEROPATHY (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3798;
To our knowledge, this is the first HSCT for XLA in developed healthcare systems solely to cure XLA or its direct complications. Patients have previously been cured of XLA through HSCT, but only when HSCT was offered for a haematological co-morbidity (e.g. Sickle Cell Disease, Leukaemia).
- Campath (alemtuzumab) / Sanofi
[VIRTUAL] HAEMATOPOIETIC STEM CELL TRANSPLANTATION FOR X-LINKED AGAMMAGLOBULINAEMIA COMPLICATED BY CHRONIC NOROVIRUS ENTEROPATHY (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3797;
To our knowledge, this is the first HSCT for XLA in developed healthcare systems solely to cure XLA or its direct complications. Patients have previously been cured of XLA through HSCT, but only when HSCT was offered for a haematological co-morbidity (e.g. Sickle Cell Disease, Leukaemia).
- Vistide (cidofovir) / Gilead
[VIRTUAL] NECROTIZING ENTEROCOLITIS IN A TWO-MONTH-OLD SCID PATIENT AFTER BONE MARROW TRANSFUSION - SURGICAL INTERVENTION AT DAY +1: YES OR NO? (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3787;
Usually, patients present mild to clinical symptoms like diarrhea, fever and thickening of the abdominal wall which can be successfully managed conservatively. Our case however, reports on an infant presenting with rapid onset of necrotizing enterocolitis and shock without signs of GvHD, necessitating an immediate decision on how to proceed.
- Vistide (cidofovir) / Gilead
[VIRTUAL] NECROTIZING ENTEROCOLITIS IN A TWO-MONTH-OLD SCID PATIENT AFTER BONE MARROW TRANSFUSION - SURGICAL INTERVENTION AT DAY +1: YES OR NO? (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3786;
Usually, patients present mild to clinical symptoms like diarrhea, fever and thickening of the abdominal wall which can be successfully managed conservatively. Our case however, reports on an infant presenting with rapid onset of necrotizing enterocolitis and shock without signs of GvHD, necessitating an immediate decision on how to proceed.
- Vistide (cidofovir) / Gilead
[VIRTUAL] NECROTIZING ENTEROCOLITIS IN A TWO-MONTH-OLD SCID PATIENT AFTER BONE MARROW TRANSFUSION - SURGICAL INTERVENTION AT DAY +1: YES OR NO? (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3785;
Usually, patients present mild to clinical symptoms like diarrhea, fever and thickening of the abdominal wall which can be successfully managed conservatively. Our case however, reports on an infant presenting with rapid onset of necrotizing enterocolitis and shock without signs of GvHD, necessitating an immediate decision on how to proceed.
- Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
[VIRTUAL] SINGLE-CENTER EXPERIENCE OF HAPLOIDENTICAL HEMATOPOIETIC STEM CELL TRANSPLANTATION WITH TCR αβ DEPLETION IN CHILDREN WITH PRIMARY IMMUNODEFICIENCY SYNDROMES (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3781;
Most patients (74.1%) received reduced-toxicity myeloablative conditioning consisting of treosulfan (n=13) or busulfan (n=6), fludarabine and thiotepa...Fourteen patients received rituximab to reduce the risk of EBV-related posttransplantation lymphoproliferative disease...Graft versus host disease (GVHD) prophylaxis consisted of cyclosporine in 10 patients, CSA+mycophenolate mofetil in 16 patients... We conclude that use of TCR αβ depleted haploidentical transplantation ensures a high engraftment rate; good immune reconstitution; low incidence of significant aGvHD, and acceptable posttransplantation morbidity in children with a range of PIDs and should be considered in children with PIDs lacking an HLA-matched donor, when urgent HSCT is indicated.
- Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
[VIRTUAL] SINGLE-CENTER EXPERIENCE OF HAPLOIDENTICAL HEMATOPOIETIC STEM CELL TRANSPLANTATION WITH TCR αβ DEPLETION IN CHILDREN WITH PRIMARY IMMUNODEFICIENCY SYNDROMES (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3780;
Most patients (74.1%) received reduced-toxicity myeloablative conditioning consisting of treosulfan (n=13) or busulfan (n=6), fludarabine and thiotepa...Fourteen patients received rituximab to reduce the risk of EBV-related posttransplantation lymphoproliferative disease...Graft versus host disease (GVHD) prophylaxis consisted of cyclosporine in 10 patients, CSA+mycophenolate mofetil in 16 patients... We conclude that use of TCR αβ depleted haploidentical transplantation ensures a high engraftment rate; good immune reconstitution; low incidence of significant aGvHD, and acceptable posttransplantation morbidity in children with a range of PIDs and should be considered in children with PIDs lacking an HLA-matched donor, when urgent HSCT is indicated.
- Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
[VIRTUAL] SINGLE-CENTER EXPERIENCE OF HAPLOIDENTICAL HEMATOPOIETIC STEM CELL TRANSPLANTATION WITH TCR αβ DEPLETION IN CHILDREN WITH PRIMARY IMMUNODEFICIENCY SYNDROMES (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3779;
Most patients (74.1%) received reduced-toxicity myeloablative conditioning consisting of treosulfan (n=13) or busulfan (n=6), fludarabine and thiotepa...Fourteen patients received rituximab to reduce the risk of EBV-related posttransplantation lymphoproliferative disease...Graft versus host disease (GVHD) prophylaxis consisted of cyclosporine in 10 patients, CSA+mycophenolate mofetil in 16 patients... We conclude that use of TCR αβ depleted haploidentical transplantation ensures a high engraftment rate; good immune reconstitution; low incidence of significant aGvHD, and acceptable posttransplantation morbidity in children with a range of PIDs and should be considered in children with PIDs lacking an HLA-matched donor, when urgent HSCT is indicated.
- Ovastat (treosulfan) / Medac, busulfan / Generic mfg.
[VIRTUAL] IMPROVED TRANSPLANT SURVIVAL AND LONG-TERM DISEASE OUTCOME FOR CHILDREN WITH CD40 LIGAND DEFICIENCY (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3769;
Survival after matched unrelated and haploidentical donors were excellent. Long-term disease outcome of survivors is good.
- Ovastat (treosulfan) / Medac, busulfan / Generic mfg.
[VIRTUAL] IMPROVED TRANSPLANT SURVIVAL AND LONG-TERM DISEASE OUTCOME FOR CHILDREN WITH CD40 LIGAND DEFICIENCY (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3768;
Survival after matched unrelated and haploidentical donors were excellent. Long-term disease outcome of survivors is good.
- Ovastat (treosulfan) / Medac, busulfan / Generic mfg.
[VIRTUAL] IMPROVED TRANSPLANT SURVIVAL AND LONG-TERM DISEASE OUTCOME FOR CHILDREN WITH CD40 LIGAND DEFICIENCY (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3767;
Survival after matched unrelated and haploidentical donors were excellent. Long-term disease outcome of survivors is good.
- [VIRTUAL] THIOTEPA VERSUS MELPHALAN CONTAINING CONDITIONING REGIMENS IN HEMATOPOIETIC STEM CELL TRANSPLANTATIONS WITH TCRαβ/CD19 GRAFT DEPLETION FOR PRIMARY IMMUNODEFICIENCIES (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3766;
No significant differences were found between the results of TCRαβ/CD19 depleted HSCT with treosulfan based CR containing melphalan and thiotepa. However, long term toxicity needs to be estimated in longer follow up.
- [VIRTUAL] THIOTEPA VERSUS MELPHALAN CONTAINING CONDITIONING REGIMENS IN HEMATOPOIETIC STEM CELL TRANSPLANTATIONS WITH TCRαβ/CD19 GRAFT DEPLETION FOR PRIMARY IMMUNODEFICIENCIES (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3765;
No significant differences were found between the results of TCRαβ/CD19 depleted HSCT with treosulfan based CR containing melphalan and thiotepa. However, long term toxicity needs to be estimated in longer follow up.
- [VIRTUAL] THIOTEPA VERSUS MELPHALAN CONTAINING CONDITIONING REGIMENS IN HEMATOPOIETIC STEM CELL TRANSPLANTATIONS WITH TCRαβ/CD19 GRAFT DEPLETION FOR PRIMARY IMMUNODEFICIENCIES (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3764;
No significant differences were found between the results of TCRαβ/CD19 depleted HSCT with treosulfan based CR containing melphalan and thiotepa. However, long term toxicity needs to be estimated in longer follow up.
- Campath (alemtuzumab) / Sanofi
[VIRTUAL] IMMUNODEFICIENCY - CENTROMERIC INSTABILITY - FACIAL DYSMORPHISM (ICF) SYNDROME: EVALUATION OF CHARACTERISTICS AND OUTCOME AFTER ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3763;
We show acceptable GVHD rates and good immune recovery. Therapy-related mortality occurred mainly in the most vulnerable patients with active infections, organ damage and/or malignancy, pointing at possible advantages of earlier HSCT in these patients.
- Campath (alemtuzumab) / Sanofi
[VIRTUAL] IMMUNODEFICIENCY - CENTROMERIC INSTABILITY - FACIAL DYSMORPHISM (ICF) SYNDROME: EVALUATION OF CHARACTERISTICS AND OUTCOME AFTER ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3762;
We show acceptable GVHD rates and good immune recovery. Therapy-related mortality occurred mainly in the most vulnerable patients with active infections, organ damage and/or malignancy, pointing at possible advantages of earlier HSCT in these patients.
- Campath (alemtuzumab) / Sanofi
[VIRTUAL] IMMUNODEFICIENCY - CENTROMERIC INSTABILITY - FACIAL DYSMORPHISM (ICF) SYNDROME: EVALUATION OF CHARACTERISTICS AND OUTCOME AFTER ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3761;
We show acceptable GVHD rates and good immune recovery. Therapy-related mortality occurred mainly in the most vulnerable patients with active infections, organ damage and/or malignancy, pointing at possible advantages of earlier HSCT in these patients.
- [VIRTUAL] COMBINED UMBILICAL CORD BLOOD AND BONE MARROW FROM HLA-IDENTICAL SIBLING DONORS FOR HEMATOPOIETIC STEM CELL TRANSPLANTATION IN CHILDREN WITH THALASSEMIA MAJOR (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3739;
A combined CB and BM graft from an HLA-identical sibling donor is an effective treatment option for β-TM in children, with less acute and chronic GVHD and lower cases of graft failure. Clinical Trial Registry: Not Applicable
- [VIRTUAL] COMBINED UMBILICAL CORD BLOOD AND BONE MARROW FROM HLA-IDENTICAL SIBLING DONORS FOR HEMATOPOIETIC STEM CELL TRANSPLANTATION IN CHILDREN WITH THALASSEMIA MAJOR (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3738;
A combined CB and BM graft from an HLA-identical sibling donor is an effective treatment option for β-TM in children, with less acute and chronic GVHD and lower cases of graft failure. Clinical Trial Registry: Not Applicable
- [VIRTUAL] COMBINED UMBILICAL CORD BLOOD AND BONE MARROW FROM HLA-IDENTICAL SIBLING DONORS FOR HEMATOPOIETIC STEM CELL TRANSPLANTATION IN CHILDREN WITH THALASSEMIA MAJOR (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3737;
A combined CB and BM graft from an HLA-identical sibling donor is an effective treatment option for β-TM in children, with less acute and chronic GVHD and lower cases of graft failure. Clinical Trial Registry: Not Applicable
- Ovastat (treosulfan) / Medac, fludarabine IV / Generic mfg., thiotepa / Generic mfg.
[VIRTUAL] A PHASE II STRATIFIED TRIAL TO ASSESS HAPLOIDENTICAL T-DEPLETED STEM CELL TRANSPLANTATION IN PATIENTS WITH SICKLE CELL DISEASE WITH NO AVAILABLE SIBLING DONOR (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3736;
Chemotherapy consists of thiotepa 10mg/kg, fludarabine 160mg/m2 and treosulfan 42g/m2, given between days -10 and -2. The objective of this trial is to prove that EFS following T-Haplo-SCT is non-inferior to MSD HSCT as well as evaluation of safety/tolerability and feasibility of haploidentical PBSC grafts depleted of TCRaβ and CD19 cells in adult and paediatric patients with sickle cell disease Clinical Trial Registry: EudraCT number: 2018-002652-337.
- Ovastat (treosulfan) / Medac, fludarabine IV / Generic mfg., thiotepa / Generic mfg.
[VIRTUAL] A PHASE II STRATIFIED TRIAL TO ASSESS HAPLOIDENTICAL T-DEPLETED STEM CELL TRANSPLANTATION IN PATIENTS WITH SICKLE CELL DISEASE WITH NO AVAILABLE SIBLING DONOR (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3735;
Chemotherapy consists of thiotepa 10mg/kg, fludarabine 160mg/m2 and treosulfan 42g/m2, given between days -10 and -2. The objective of this trial is to prove that EFS following T-Haplo-SCT is non-inferior to MSD HSCT as well as evaluation of safety/tolerability and feasibility of haploidentical PBSC grafts depleted of TCRaβ and CD19 cells in adult and paediatric patients with sickle cell disease Clinical Trial Registry: EudraCT number: 2018-002652-337.
- Ovastat (treosulfan) / Medac, fludarabine IV / Generic mfg., thiotepa / Generic mfg.
[VIRTUAL] A PHASE II STRATIFIED TRIAL TO ASSESS HAPLOIDENTICAL T-DEPLETED STEM CELL TRANSPLANTATION IN PATIENTS WITH SICKLE CELL DISEASE WITH NO AVAILABLE SIBLING DONOR (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3734;
Chemotherapy consists of thiotepa 10mg/kg, fludarabine 160mg/m2 and treosulfan 42g/m2, given between days -10 and -2. The objective of this trial is to prove that EFS following T-Haplo-SCT is non-inferior to MSD HSCT as well as evaluation of safety/tolerability and feasibility of haploidentical PBSC grafts depleted of TCRaβ and CD19 cells in adult and paediatric patients with sickle cell disease Clinical Trial Registry: EudraCT number: 2018-002652-337.
- Campath (alemtuzumab) / Sanofi, cyclophosphamide intravenous / Generic mfg.
[VIRTUAL] HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR SICKLE CELL DISEASE: THE SPANISH EXPERIENCE (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3724;
SCD represents a chronic and multisystemic disease with a high morbidity and mortality. Results with MSD transplant are excellent; therefore it is important to refer these patients to centers with experience and promptly assess the indication of HSCT.
- Campath (alemtuzumab) / Sanofi, cyclophosphamide intravenous / Generic mfg.
[VIRTUAL] HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR SICKLE CELL DISEASE: THE SPANISH EXPERIENCE (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3723;
SCD represents a chronic and multisystemic disease with a high morbidity and mortality. Results with MSD transplant are excellent; therefore it is important to refer these patients to centers with experience and promptly assess the indication of HSCT.
- Campath (alemtuzumab) / Sanofi, cyclophosphamide intravenous / Generic mfg.
[VIRTUAL] HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR SICKLE CELL DISEASE: THE SPANISH EXPERIENCE (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3722;
SCD represents a chronic and multisystemic disease with a high morbidity and mortality. Results with MSD transplant are excellent; therefore it is important to refer these patients to centers with experience and promptly assess the indication of HSCT.
- Campath (alemtuzumab) / Sanofi, tacrolimus / Generic mfg., cyclophosphamide intravenous / Generic mfg.
[VIRTUAL] BONE MARROW TRANSPLANT IN PATIENTS WITH SICKLE CELL ANAEMIA. EXPERIENCE IN ONE CENTRE (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3709;
The outcome of our center, the largest HCST series in Spain is similar to the international cohort and confirms the role of HLA-identical sibling transplantation for children with SCD. Since 2015 we improved our results, with less toxicity and without mortality.
- Campath (alemtuzumab) / Sanofi, tacrolimus / Generic mfg., cyclophosphamide intravenous / Generic mfg.
[VIRTUAL] BONE MARROW TRANSPLANT IN PATIENTS WITH SICKLE CELL ANAEMIA. EXPERIENCE IN ONE CENTRE (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3708;
The outcome of our center, the largest HCST series in Spain is similar to the international cohort and confirms the role of HLA-identical sibling transplantation for children with SCD. Since 2015 we improved our results, with less toxicity and without mortality.
- Campath (alemtuzumab) / Sanofi, tacrolimus / Generic mfg., cyclophosphamide intravenous / Generic mfg.
[VIRTUAL] BONE MARROW TRANSPLANT IN PATIENTS WITH SICKLE CELL ANAEMIA. EXPERIENCE IN ONE CENTRE (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3707;
The outcome of our center, the largest HCST series in Spain is similar to the international cohort and confirms the role of HLA-identical sibling transplantation for children with SCD. Since 2015 we improved our results, with less toxicity and without mortality.
- spanlecortemlocel (MGTA-456) / Magenta Therap
[VIRTUAL] HIGH DOSE STEM CELL THERAPIES, LIKE MGTA-456, ENABLE COMPLETE NEURAL AND PERIPHERAL DISEASE CROSS-CORRECTION THROUGH RAPID AND ROBUST HEMATOPOIETIC ENGRAFTMENT (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3697;
We show that high dose HSCT leads to rapid and durable improved disease correction, including normalization of behavioral outcomes, via robust microglial engraftment. High dose cell therapies, like MGTA-456, may resolve neurologic disease in patients with IMDs and other neurodegenerative diseases caused by defective microglia.
- spanlecortemlocel (MGTA-456) / Magenta Therap
[VIRTUAL] HIGH DOSE STEM CELL THERAPIES, LIKE MGTA-456, ENABLE COMPLETE NEURAL AND PERIPHERAL DISEASE CROSS-CORRECTION THROUGH RAPID AND ROBUST HEMATOPOIETIC ENGRAFTMENT (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3696;
We show that high dose HSCT leads to rapid and durable improved disease correction, including normalization of behavioral outcomes, via robust microglial engraftment. High dose cell therapies, like MGTA-456, may resolve neurologic disease in patients with IMDs and other neurodegenerative diseases caused by defective microglia.