autologous hematopoietic stem cell therapy / Northwestern University - LARVOL DELTA

[VIRTUAL] Clinical results after autologous hematopoietic stem cell transplantation with betibeglogene autotemcel (beti-cel, LentiGlobin for ß-thalassemia) gene therapy in phase 3 Nothstar-2 and Northstar-3 studies in transfusion-dependent ß-thalassemia (TDT) (DGHO 2020) - P3 | "Introduction: In the phase 1/2 Northstar study (HGB-204) of beti-cel, patients achieving transfusion independence (TI) showed improved erythropoiesis and reductions from baseline to 48 months in median serum ferritin (4829 [n=10] to 937 [n=7] pmol/L) and liver iron levels (6.3 [n=11] to 2.0 [n=7] mg/g dw). After beti-cel gene therapy, 20/22 patients (non-β0/β0) and 11/13 patients (β0/β0, β0/β+ IVS-I-110 or β+ IVS-I-110/β+ IVS-I-110) with TDT and ≥6 months follow-up have stopped transfusions. 89% and 75% of evaluable patients in Northstar-2 and -3 achieved TI. Safety is consistent with that of single-agent busulfan myeloablation."

Clinical • P3 data • Cardiovascular • Dental Disorders • Gene Therapies • Genetic Disorders • Hepatology • Leukopenia • Neutropenia • Pain • Stomatitis • Thrombocytopenia • Transplantation • CD34

[VIRTUAL] Clinical results after autologous hematopoietic stem cell transplantation with betibeglogene autotemcel (beti-cel, LentiGlobin for ß-thalassemia) gene therapy in phase 3 Nothstar-2 and Northstar-3 studies in transfusion-dependent ß-thalassemia (TDT) (DGHO 2020) - P3 | "Introduction: In the phase 1/2 Northstar study (HGB-204) of beti-cel, patients achieving transfusion independence (TI) showed improved erythropoiesis and reductions from baseline to 48 months in median serum ferritin (4829 [n=10] to 937 [n=7] pmol/L) and liver iron levels (6.3 [n=11] to 2.0 [n=7] mg/g dw). After beti-cel gene therapy, 20/22 patients (non-β0/β0) and 11/13 patients (β0/β0, β0/β+ IVS-I-110 or β+ IVS-I-110/β+ IVS-I-110) with TDT and ≥6 months follow-up have stopped transfusions. 89% and 75% of evaluable patients in Northstar-2 and -3 achieved TI. Safety is consistent with that of single-agent busulfan myeloablation."

Clinical • P3 data • Cardiovascular • Dental Disorders • Gene Therapies • Genetic Disorders • Hepatology • Leukopenia • Neutropenia • Pain • Stomatitis • Thrombocytopenia • Transplantation • CD34

[VIRTUAL] Clinical results after autologous hematopoietic stem cell transplantation with betibeglogene autotemcel (beti-cel, LentiGlobin for ß-thalassemia) gene therapy in phase 3 Nothstar-2 and Northstar-3 studies in transfusion-dependent ß-thalassemia (TDT) (DGHO 2020) - P3 | "Introduction: In the phase 1/2 Northstar study (HGB-204) of beti-cel, patients achieving transfusion independence (TI) showed improved erythropoiesis and reductions from baseline to 48 months in median serum ferritin (4829 [n=10] to 937 [n=7] pmol/L) and liver iron levels (6.3 [n=11] to 2.0 [n=7] mg/g dw). After beti-cel gene therapy, 20/22 patients (non-β0/β0) and 11/13 patients (β0/β0, β0/β+ IVS-I-110 or β+ IVS-I-110/β+ IVS-I-110) with TDT and ≥6 months follow-up have stopped transfusions. 89% and 75% of evaluable patients in Northstar-2 and -3 achieved TI. Safety is consistent with that of single-agent busulfan myeloablation."

Clinical • P3 data • Cardiovascular • Dental Disorders • Gene Therapies • Genetic Disorders • Hepatology • Leukopenia • Neutropenia • Pain • Stomatitis • Thrombocytopenia • Transplantation • CD34

[VIRTUAL] Clinical results after autologous hematopoietic stem cell transplantation with betibeglogene autotemcel (beti-cel, LentiGlobin for ß-thalassemia) gene therapy in phase 3 Nothstar-2 and Northstar-3 studies in transfusion-dependent ß-thalassemia (TDT) (DGHO 2020) - P3 | "Introduction: In the phase 1/2 Northstar study (HGB-204) of beti-cel, patients achieving transfusion independence (TI) showed improved erythropoiesis and reductions from baseline to 48 months in median serum ferritin (4829 [n=10] to 937 [n=7] pmol/L) and liver iron levels (6.3 [n=11] to 2.0 [n=7] mg/g dw). After beti-cel gene therapy, 20/22 patients (non-β0/β0) and 11/13 patients (β0/β0, β0/β+ IVS-I-110 or β+ IVS-I-110/β+ IVS-I-110) with TDT and ≥6 months follow-up have stopped transfusions. 89% and 75% of evaluable patients in Northstar-2 and -3 achieved TI. Safety is consistent with that of single-agent busulfan myeloablation."

Clinical • P3 data • Cardiovascular • Dental Disorders • Gene Therapies • Genetic Disorders • Hepatology • Leukopenia • Neutropenia • Pain • Stomatitis • Thrombocytopenia • Transplantation • CD34

Real-world application of autologous hematopoietic stem cell transplantation in 507 patients with multiple sclerosis. (PubMed, J Neurol) - "In patients with RRMS, autologous non-myeloablative HSCT is an effective one-time therapy, while HSCT appears of less benefit for newly diagnosed SPMS."

Clinical • Journal • Real-world evidence • Bone Marrow Transplantation • CNS Disorders • Endocrine Disorders • Hematological Disorders • Immunology • Infectious Disease • Multiple Sclerosis • Pneumonia • Respiratory Diseases • Septic Shock • Thrombocytopenia • Thrombocytopenic Purpura • Transplantation

Safety and Toxicity Profile of Pembrolizumab (PEM) in Combination with ICE Chemotherapy Followed By Autologous Stem Cell Transplantation for Relapsed/Refractory Classical Hodgkin Lymphoma: No Impairment in Stem Cell Mobilization or Engraftment (ASH 2019) - P2; "Background: Despite excellent outcomes in the front-line management of classical Hodgkin lymphoma (cHL), patients with relapsed or refractory disease typically undergo second-line cytotoxic chemotherapy before proceeding to consolidation with autologous hematopoietic stem cell transplant (autoHSCT)...The PD-1 pathway plays an important role in the pathogenesis of cHL and checkpoint inhibition with agents including PEM and nivolumab have shown efficacy as monotherapy in heavily pretreated disease...Patients are treated with 2 cycles of PEM 200 mg IV on day 1 in combination with ICE chemotherapy (ifosfamide 5000 mg/m2 day 2 CIV over 24hr, carboplatin AUC 5 IV day 2, etoposide 100 mg/m2 IV days 1-3) on a Q21 day cycle...Following protocol directed therapy, all but one patient had successful mobilization and collection; one had a severe allergic reaction to filgrastim and underwent bone marrow harvest instead... The combination of PEM with ICE chemotherapy in relapsed /..."

Clinical • Combination therapy • CD34 • FDG PET

Long term outcomes of the French ASTIS systemic sclerosis cohort using the global rank composite score. (PubMed, Bone Marrow Transplant) - "Two randomised trials (ASTIS, SCOT) of Autologous Hematopoietic Stem Cell Transplantation (AHSCT) versus monthly Cyclophosphamide for severe Systemic Sclerosis (SSc) patients used similar inclusion criteria, but different primary endpoints: event-free-survival (EFS) at 24 months in ASTIS versus the global rank composite score (GRCS) at 54 months in SCOT...In intention-to-treat analysis, the GRCS demonstrated superiority for AHSCT (median: 9 versus -19, p = 0.018), mRSS (Δ mRSS: -16 versus -9, p = 0.02), and HAQ-DI (ΔHAQ-DI: -0.89 versus -0.2, p = 0.05) with no significant difference in OS, EFS, lung, heart and kidney function between the groups. In conclusion, this study demonstrates long term benefits of non-myeloablative AHSCT when assessed by the five longitudinal measures within GRCS affording direct primary endpoint comparison between ASTIS and SCOT."

Clinical • Journal • Bone Marrow Transplantation • Fibrosis • Immunology • Scleroderma • Systemic Sclerosis • Transplantation

Harnessing Tolerogenic Histone Peptide Epitopes From Nucleosomes for Selective Down-Regulation of Pathogenic Autoimmune Response in Lupus (Past, Present, and Future). (PubMed, Front Immunol) - "Furthermore, the same types of Treg cells are generated in lupus patients who are in very long-term remission (2-8 years) after undergoing autologous hematopoietic stem cell transplantation...The histone peptide tolerance is a natural and non-toxic therapy suitable for treating early lupus, and also maintaining lupus patients after toxic drug therapy. The experimental steps, challenges and possible solutions for successful therapy with these peptide epitopes are discussed in this highly focused review on Systemic Lupus."

Journal • Review • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus • Transplantation • CD4 • CD8 • FOXP3 • IL2RA • IL6 • TGFB1

New autoimmune diseases after autologous hematopoietic stem cell transplantation for multiple sclerosis. (PubMed, Bone Marrow Transplant) - "Secondary autoimmune diseases (2ndADs), most frequently autoimmune cytopenias (AICs), were first described after allogeneic hematopoietic stem cell transplantation (HSCT) undertaken for malignant and hematological indications, occurred at a prevalence of ~5-6.5%, and were attributed to allogeneic immune imbalances in the context of graft versus host disease, viral infections, and chronic immunosuppression. Alemtuzumab in the conditioning regimen has been identified as a risk for development of 2ndADs after either allogeneic or autologous HSCT and is consistent with the high rates of 2ndADs when using alemtuzumab as monotherapy. Due to the significant consequences but variable incidence, depending on conditioning regimen, of 2ndADs and similarity in known immune reconstitution kinetics after autologous HSCT for autoimmune diseases and after alemtuzumab monotherapy, we propose that an imbalance between B and T lineage regeneration early after HSCT may underlie the..."

Journal • Review • CNS Disorders • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Multiple Sclerosis • Transplantation

[VIRTUAL] OUTCOME OF NON-MYELOABLATIVE AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR MULTIPLE SCLEROSIS: A SINGLE CENTER SUMMARY OF 511 PATIENTS (EBMT 2021) - " Five hundred and eleven patients at Northwestern University between 7-2003 and 10-2019 underwent non-myeloablative HSCT using a non-selected graft and cyclophosphamide (Cy) / rabbit anti-thymocyte globulin (ATG) (n=376), Cy / ATG / rituximab (n=63), Cy / ATG / intravenous immunoglobulin (IVIG) (n=46), or Cy / alemtuzumab (n=26). Either an intravenous cephalosporin cefepime (maxipimeâ) or piperacillin / tozobactam (zosyn®) was started on day 0 and continued until engraftment that usually occurred on day 9 or 10... Autologous non-myeloablative HSCT provides drug free reversal of neurologic disability of greater than 1.0 EDSS points for greater than 5 years in 89% of patients with RRMS. The low incidence of bacteremia may be related to the use of non-myeloablative regimens that do not cause mucositis, i.e. gut endothelium bacterial barrier remains closed, placement and removal of the large bore apheresis catheter on the same day and subsequent insertion..."

Clinical • Cardiovascular • CNS Disorders • Colon Cancer • Colorectal Cancer • Endocrine Disorders • Gastrointestinal Cancer • Hematological Disorders • Immunology • Infectious Disease • Mucositis • Multiple Sclerosis • Myocardial Infarction • Neutropenia • Oncology • Pneumonia • Respiratory Diseases • Solid Tumor • Thrombocytopenia • Thrombocytopenic Purpura • Transplantation

[VIRTUAL] OUTCOME OF NON-MYELOABLATIVE AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR MULTIPLE SCLEROSIS: A SINGLE CENTER SUMMARY OF 511 PATIENTS (EBMT 2021) - " Five hundred and eleven patients at Northwestern University between 7-2003 and 10-2019 underwent non-myeloablative HSCT using a non-selected graft and cyclophosphamide (Cy) / rabbit anti-thymocyte globulin (ATG) (n=376), Cy / ATG / rituximab (n=63), Cy / ATG / intravenous immunoglobulin (IVIG) (n=46), or Cy / alemtuzumab (n=26). Either an intravenous cephalosporin cefepime (maxipimeâ) or piperacillin / tozobactam (zosyn®) was started on day 0 and continued until engraftment that usually occurred on day 9 or 10... Autologous non-myeloablative HSCT provides drug free reversal of neurologic disability of greater than 1.0 EDSS points for greater than 5 years in 89% of patients with RRMS. The low incidence of bacteremia may be related to the use of non-myeloablative regimens that do not cause mucositis, i.e. gut endothelium bacterial barrier remains closed, placement and removal of the large bore apheresis catheter on the same day and subsequent insertion..."

Clinical • Cardiovascular • CNS Disorders • Colon Cancer • Colorectal Cancer • Endocrine Disorders • Gastrointestinal Cancer • Hematological Disorders • Immunology • Infectious Disease • Mucositis • Multiple Sclerosis • Myocardial Infarction • Neutropenia • Oncology • Pneumonia • Respiratory Diseases • Solid Tumor • Thrombocytopenia • Thrombocytopenic Purpura • Transplantation

[VIRTUAL] OUTCOME OF NON-MYELOABLATIVE AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR MULTIPLE SCLEROSIS: A SINGLE CENTER SUMMARY OF 511 PATIENTS (EBMT 2021) - " Five hundred and eleven patients at Northwestern University between 7-2003 and 10-2019 underwent non-myeloablative HSCT using a non-selected graft and cyclophosphamide (Cy) / rabbit anti-thymocyte globulin (ATG) (n=376), Cy / ATG / rituximab (n=63), Cy / ATG / intravenous immunoglobulin (IVIG) (n=46), or Cy / alemtuzumab (n=26). Either an intravenous cephalosporin cefepime (maxipimeâ) or piperacillin / tozobactam (zosyn®) was started on day 0 and continued until engraftment that usually occurred on day 9 or 10... Autologous non-myeloablative HSCT provides drug free reversal of neurologic disability of greater than 1.0 EDSS points for greater than 5 years in 89% of patients with RRMS. The low incidence of bacteremia may be related to the use of non-myeloablative regimens that do not cause mucositis, i.e. gut endothelium bacterial barrier remains closed, placement and removal of the large bore apheresis catheter on the same day and subsequent insertion..."

Clinical • Cardiovascular • CNS Disorders • Colon Cancer • Colorectal Cancer • Endocrine Disorders • Gastrointestinal Cancer • Hematological Disorders • Immunology • Infectious Disease • Mucositis • Multiple Sclerosis • Myocardial Infarction • Neutropenia • Oncology • Pneumonia • Respiratory Diseases • Solid Tumor • Thrombocytopenia • Thrombocytopenic Purpura • Transplantation

[VIRTUAL] OUTCOME OF NON-MYELOABLATIVE AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR MULTIPLE SCLEROSIS: A SINGLE CENTER SUMMARY OF 511 PATIENTS (EBMT 2021) - " Five hundred and eleven patients at Northwestern University between 7-2003 and 10-2019 underwent non-myeloablative HSCT using a non-selected graft and cyclophosphamide (Cy) / rabbit anti-thymocyte globulin (ATG) (n=376), Cy / ATG / rituximab (n=63), Cy / ATG / intravenous immunoglobulin (IVIG) (n=46), or Cy / alemtuzumab (n=26). Either an intravenous cephalosporin cefepime (maxipimeâ) or piperacillin / tozobactam (zosyn®) was started on day 0 and continued until engraftment that usually occurred on day 9 or 10... Autologous non-myeloablative HSCT provides drug free reversal of neurologic disability of greater than 1.0 EDSS points for greater than 5 years in 89% of patients with RRMS. The low incidence of bacteremia may be related to the use of non-myeloablative regimens that do not cause mucositis, i.e. gut endothelium bacterial barrier remains closed, placement and removal of the large bore apheresis catheter on the same day and subsequent insertion..."

Clinical • Cardiovascular • CNS Disorders • Colon Cancer • Colorectal Cancer • Endocrine Disorders • Gastrointestinal Cancer • Hematological Disorders • Immunology • Infectious Disease • Mucositis • Multiple Sclerosis • Myocardial Infarction • Neutropenia • Oncology • Pneumonia • Respiratory Diseases • Solid Tumor • Thrombocytopenia • Thrombocytopenic Purpura • Transplantation

[VIRTUAL] High-Dose Carboplatin and Thiotepa with Autologous Hematopoietic Stem Cell Rescue (auHSCR) for Medulloblastoma (Mdl) and Atypical Teratoid Rhabdoid Tumors (ATRT) of Central Nervous System (CNS) (TCT-ASTCT-CIBMTR 2021) - "Post-relapse therapies may offer a chance for long-term survival in Mdl patients, particularly if irradiation therapy remains an option. Long-term follow-up will be essential for assessing the quality of life after this regimen."

Medulloblastoma • Oncology • Rhabdoid Tumor • Sarcoma • Solid Tumor

[VIRTUAL] High-Dose Carboplatin and Thiotepa with Autologous Hematopoietic Stem Cell Rescue (auHSCR) for Medulloblastoma (Mdl) and Atypical Teratoid Rhabdoid Tumors (ATRT) of Central Nervous System (CNS) (TCT-ASTCT-CIBMTR 2021) - "Post-relapse therapies may offer a chance for long-term survival in Mdl patients, particularly if irradiation therapy remains an option. Long-term follow-up will be essential for assessing the quality of life after this regimen."

Medulloblastoma • Oncology • Rhabdoid Tumor • Sarcoma • Solid Tumor

[VIRTUAL] High-Dose Carboplatin and Thiotepa with Autologous Hematopoietic Stem Cell Rescue (auHSCR) for Medulloblastoma (Mdl) and Atypical Teratoid Rhabdoid Tumors (ATRT) of Central Nervous System (CNS) (TCT-ASTCT-CIBMTR 2021) - "Post-relapse therapies may offer a chance for long-term survival in Mdl patients, particularly if irradiation therapy remains an option. Long-term follow-up will be essential for assessing the quality of life after this regimen."

Medulloblastoma • Oncology • Rhabdoid Tumor • Sarcoma • Solid Tumor

[VIRTUAL] High-Dose Carboplatin and Thiotepa with Autologous Hematopoietic Stem Cell Rescue (auHSCR) for Medulloblastoma (Mdl) and Atypical Teratoid Rhabdoid Tumors (ATRT) of Central Nervous System (CNS) (TCT-ASTCT-CIBMTR 2021) - "Post-relapse therapies may offer a chance for long-term survival in Mdl patients, particularly if irradiation therapy remains an option. Long-term follow-up will be essential for assessing the quality of life after this regimen."

Medulloblastoma • Oncology • Rhabdoid Tumor • Sarcoma • Solid Tumor

[VIRTUAL] High-Dose Carboplatin and Thiotepa with Autologous Hematopoietic Stem Cell Rescue (auHSCR) for Medulloblastoma (Mdl) and Atypical Teratoid Rhabdoid Tumors (ATRT) of Central Nervous System (CNS) (TCT-ASTCT-CIBMTR 2021) - "Post-relapse therapies may offer a chance for long-term survival in Mdl patients, particularly if irradiation therapy remains an option. Long-term follow-up will be essential for assessing the quality of life after this regimen."

Medulloblastoma • Oncology • Rhabdoid Tumor • Sarcoma • Solid Tumor

Stem Cell Transplantation for Stiff Person Syndrome (SPS) (clinicaltrials.gov) - P1/2; N=23; Terminated; Sponsor: Northwestern University; Active, not recruiting ➔ Terminated; Could not predict who would respond, relapse or go into remission

Clinical • Trial termination • Ataxia • CNS Disorders • Immunology • Movement Disorders • Transplantation

[VIRTUAL] A PILOT FEASIBILITY STUDY OF NON-MYELOABLATIVE ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR REFRACTORY CROHN DISEASE (EBMT 2020) - "Background: Autologous hematopoietic stem cell transplantation (HSCT) induces remission of refractory Crohn disease (CD) but approximately 50% and 80% relapse and restart standard therapies at 3 and 5 years, respectively, post HSCT... Eligible patients were 18 to 45 years old, had an established diagnosis of Crohn disease, failed corticosteroids, 5-aminosalicylate, and at least two anti-TNF (infliximab, adalimumab, or certolizumab) drugs with a Crohn disease activity index (CDAI) between 250 to 400 or Craig Severity Score (CSS) > 17...Conditioning drugs were: 1) cyclophosphamide 200 mg/kg divided as 50 mg /kg on days -5,-4,-3 and -2, 2) fludarabine (75 to 150 mg / m2) divided as 25- 30 mg /m2 /day (between days -7 to -3), and alemtuzumab (60 mg to 90 mg) divided 30 mg day (between days -3 to -1). Cyclosporine (100 mg po bid) or tacrolimus (1.0 mg bid) was initiated on day -2 and continued for 9 months... Complete treatment-free 5-year remissions without donor..."

Acute Kidney Injury • Chronic Kidney Disease • Crohn's disease • Gastroenterology • Genetic Disorders • Graft versus Host Disease • Immunology • Infectious Disease • Inflammatory Bowel Disease • Neutropenia • Septic Shock • Transplantation • CD33

CAST: Cardiac Safe Transplants for Systemic Sclerosis (clinicaltrials.gov) - P2/3; N=9; Terminated; Sponsor: Northwestern University; N=50 ➔ 9; Trial completion date: Jan 2026 ➔ Oct 2019; Active, not recruiting ➔ Terminated; Trial primary completion date: Jan 2025 ➔ Oct 2019; PI Sabbatical

Clinical • Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Fibrosis • Immunology • Scleroderma • Systemic Sclerosis • Transplantation

[VIRTUAL] A PILOT FEASIBILITY STUDY OF NON-MYELOABLATIVE ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR REFRACTORY CROHN DISEASE (EBMT 2020) - "Background: Autologous hematopoietic stem cell transplantation (HSCT) induces remission of refractory Crohn disease (CD) but approximately 50% and 80% relapse and restart standard therapies at 3 and 5 years, respectively, post HSCT... Eligible patients were 18 to 45 years old, had an established diagnosis of Crohn disease, failed corticosteroids, 5-aminosalicylate, and at least two anti-TNF (infliximab, adalimumab, or certolizumab) drugs with a Crohn disease activity index (CDAI) between 250 to 400 or Craig Severity Score (CSS) > 17...Conditioning drugs were: 1) cyclophosphamide 200 mg/kg divided as 50 mg /kg on days -5,-4,-3 and -2, 2) fludarabine (75 to 150 mg / m2) divided as 25- 30 mg /m2 /day (between days -7 to -3), and alemtuzumab (60 mg to 90 mg) divided 30 mg day (between days -3 to -1). Cyclosporine (100 mg po bid) or tacrolimus (1.0 mg bid) was initiated on day -2 and continued for 9 months... Complete treatment-free 5-year remissions without donor..."

Acute Kidney Injury • Chronic Kidney Disease • Crohn's disease • Gastroenterology • Genetic Disorders • Graft versus Host Disease • Immunology • Infectious Disease • Inflammatory Bowel Disease • Neutropenia • Septic Shock • Transplantation • CD33

[VIRTUAL] A PILOT FEASIBILITY STUDY OF NON-MYELOABLATIVE ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR REFRACTORY CROHN DISEASE (EBMT 2020) - "Background: Autologous hematopoietic stem cell transplantation (HSCT) induces remission of refractory Crohn disease (CD) but approximately 50% and 80% relapse and restart standard therapies at 3 and 5 years, respectively, post HSCT... Eligible patients were 18 to 45 years old, had an established diagnosis of Crohn disease, failed corticosteroids, 5-aminosalicylate, and at least two anti-TNF (infliximab, adalimumab, or certolizumab) drugs with a Crohn disease activity index (CDAI) between 250 to 400 or Craig Severity Score (CSS) > 17...Conditioning drugs were: 1) cyclophosphamide 200 mg/kg divided as 50 mg /kg on days -5,-4,-3 and -2, 2) fludarabine (75 to 150 mg / m2) divided as 25- 30 mg /m2 /day (between days -7 to -3), and alemtuzumab (60 mg to 90 mg) divided 30 mg day (between days -3 to -1). Cyclosporine (100 mg po bid) or tacrolimus (1.0 mg bid) was initiated on day -2 and continued for 9 months... Complete treatment-free 5-year remissions without donor..."

Acute Kidney Injury • Chronic Kidney Disease • Crohn's disease • Gastroenterology • Genetic Disorders • Graft versus Host Disease • Immunology • Infectious Disease • Inflammatory Bowel Disease • Neutropenia • Septic Shock • Transplantation • CD33

[VIRTUAL] A PILOT FEASIBILITY STUDY OF NON-MYELOABLATIVE ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR REFRACTORY CROHN DISEASE (EBMT 2020) - "Background: Autologous hematopoietic stem cell transplantation (HSCT) induces remission of refractory Crohn disease (CD) but approximately 50% and 80% relapse and restart standard therapies at 3 and 5 years, respectively, post HSCT... Eligible patients were 18 to 45 years old, had an established diagnosis of Crohn disease, failed corticosteroids, 5-aminosalicylate, and at least two anti-TNF (infliximab, adalimumab, or certolizumab) drugs with a Crohn disease activity index (CDAI) between 250 to 400 or Craig Severity Score (CSS) > 17...Conditioning drugs were: 1) cyclophosphamide 200 mg/kg divided as 50 mg /kg on days -5,-4,-3 and -2, 2) fludarabine (75 to 150 mg / m2) divided as 25- 30 mg /m2 /day (between days -7 to -3), and alemtuzumab (60 mg to 90 mg) divided 30 mg day (between days -3 to -1). Cyclosporine (100 mg po bid) or tacrolimus (1.0 mg bid) was initiated on day -2 and continued for 9 months... Complete treatment-free 5-year remissions without donor..."

Acute Kidney Injury • Chronic Kidney Disease • Crohn's disease • Gastroenterology • Genetic Disorders • Graft versus Host Disease • Immunology • Infectious Disease • Inflammatory Bowel Disease • Neutropenia • Septic Shock • Transplantation • CD33

[VIRTUAL] CARDIAC SAFE HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR SYSTEMIC SCLEROSIS WITH COMPROMISED CARDIAC FUNCTION (EBMT 2020) - "Background: While three randomized trials have demonstrated superiority of autologous hematopoietic stem cell transplantation (HSCT) over monthly intravenous cyclophosphamide for systemic sclerosis, transplantation has been complicated by 6-10% treatment related mortality...Peripheral blood stem cells were collected 10 days after intravenous cyclophosphamide (2 g/m2) and 5 to 10 µg/kg per day of subcutaneous filgrastim beginning 5 days after cyclophosphamide...Of 15 patients treated with fludarabine-based regimen , nine (60%) are alive and improving, two died within one year from disease progression, one did not respond but is alive, and three others restarted maintenance daily cellcept for skin tightening... HSCT using a regimen based on fludarabine 120 mg, cyclophosphamide (60 mg), rATG (6.0 mg/kg) with or without rituximab (500 or 1000 mg) is well tolerated in patients with SSc and compromised cardiac function. However, relapse is high without the addition of..."

Cardiovascular • Congestive Heart Failure • Heart Failure • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Neutropenia • Oncology • Scleroderma • Solid Tumor • Systemic Sclerosis • Transplantation • MRI