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Publication Record


Low-Dose Anti-Thymocyte Globulin (ATG) Preserves β-Cell Function and Improves HbA in New-Onset Type 1 Diabetes.
Haller MJ, Schatz DA, Skyler JS, Krischer JP, Bundy BN, Miller JL, Atkinson MA, Becker DJ, Baidal D, DiMeglio LA, Gitelman SE, Goland R, Gottlieb PA, Herold KC, Marks JB, Moran A, Rodriguez H, Russell W, Wilson DM, Greenbaum CJ, Type 1 Diabetes TrialNet ATG-GCSF Study Group
(2018) Diabetes Care 41: 1917-1925
MeSH Terms: Adolescent, Adult, Antilymphocyte Serum, C-Peptide, Child, Cytoprotection, Diabetes Mellitus, Type 1, Dose-Response Relationship, Drug, Double-Blind Method, Drug Therapy, Combination, Female, Glycated Hemoglobin A, Granulocyte Colony-Stimulating Factor, Humans, Insulin-Secreting Cells, Male, Pilot Projects, Polyethylene Glycols, Recombinant Proteins, Young Adult
Show Abstract · Added May 2, 2019
OBJECTIVE - A pilot study suggested that combination therapy with low-dose anti-thymocyte globulin (ATG) and pegylated granulocyte colony-stimulating factor (GCSF) preserves C-peptide in established type 1 diabetes (T1D) (duration 4 months to 2 years). We hypothesized that ) low-dose ATG/GCSF or ) low-dose ATG alone would slow the decline of β-cell function in patients with new-onset T1D (duration <100 days).
RESEARCH DESIGN AND METHODS - A three-arm, randomized, double-masked, placebo-controlled trial was performed by the Type 1 Diabetes TrialNet Study Group in 89 subjects: 29 subjects randomized to ATG (2.5 mg/kg intravenously) followed by pegylated GCSF (6 mg subcutaneously every 2 weeks for 6 doses), 29 to ATG alone (2.5 mg/kg), and 31 to placebo. The primary end point was mean area under the curve (AUC) C-peptide during a 2-h mixed-meal tolerance test 1 year after initiation of therapy. Significance was defined as one-sided value < 0.025.
RESULTS - The 1-year mean AUC C-peptide was significantly higher in subjects treated with ATG (0.646 nmol/L) versus placebo (0.406 nmol/L) ( = 0.0003) but not in those treated with ATG/GCSF (0.528 nmol/L) versus placebo ( = 0.031). HbA was significantly reduced at 1 year in subjects treated with ATG and ATG/GCSF, = 0.002 and 0.011, respectively.
CONCLUSIONS - Low-dose ATG slowed decline of C-peptide and reduced HbA in new-onset T1D. Addition of GCSF did not enhance C-peptide preservation afforded by low-dose ATG. Future studies should be considered to determine whether low-dose ATG alone or in combination with other agents may prevent or delay the onset of the disease.
© 2018 by the American Diabetes Association.
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MeSH Terms
Targeted Busulfan therapy with a steady-state concentration of 600-700 ng/mL in patients with sickle cell disease receiving HLA-identical sibling bone marrow transplant.
Maheshwari S, Kassim A, Yeh RF, Domm J, Calder C, Evans M, Manes B, Bruce K, Brown V, Ho R, Frangoul H, Yang E
(2014) Bone Marrow Transplant 49: 366-9
MeSH Terms: Adolescent, Age Factors, Anemia, Sickle Cell, Antilymphocyte Serum, Bone Marrow Transplantation, Busulfan, Child, Child, Preschool, Cyclophosphamide, Disease-Free Survival, Female, Follow-Up Studies, HLA Antigens, Humans, Immunosuppressive Agents, Infant, Male, Neutrophils, Siblings, Treatment Outcome
Show Abstract · Added March 10, 2014
Busulfan (BU) has a narrow therapeutic window and the average concentration of BU at steady state (Css) is critical for successful engraftment in children receiving BU as part of the preparative regimen for allogeneic transplants. Sixteen patients with sickle cell disease (SCD) underwent allogeneic bone marrow transplant (BMT) from HLA-identical siblings. The preparative regimen consisted of intravenous BU 0.8-1 mg/kg/dose for 16 doses, cytoxan (CY) of 50 mg/kg daily for four doses and equine anti-thymocyte globulin (ATG) 30 mg/kg daily for three doses. BU levels were adjusted to provide a total exposure Css of 600-700 ng/mL. The median age at the time of transplant was 6.2 years (range 1.2-19.3). Fourteen (87%) patients required adjustment of the BU dose to achieve a median Css of 652 ng/mL (range 607-700). All patients achieved neutrophil and platelet engraftment without significant toxicity. Median donor engraftment at the last follow-up was 100% (range 80-100). None of the patients experienced sickle cell-related complications post transplant. With a median follow-up of 3 years (range 1.3-9), the event-free survival (EFS) and overall survival (OS) are both 100%. We conclude that targeting of BU Css between 600 and 700 ng/mL in this regimen can result in excellent and sustained engraftment in young patients with SCD.
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20 MeSH Terms
Renal transplantation in the setting of early steroid withdrawal: a comparison of rabbit antithymocyte globulin induction dosing in two eras.
Rogers CC, Asipenko N, Horwedel T, Gautam S, Goldfarb-Rumyantzev AS, Pavlakis M, Johnson SR, Karp SJ, Evenson A, Khwaja K, Hanto DW, Mandelbrot DA
(2013) Am J Nephrol 38: 397-404
MeSH Terms: Aged, Animals, Antilymphocyte Serum, Drug Administration Schedule, Female, Graft Rejection, Graft Survival, Humans, Immunosuppression, Immunosuppressive Agents, Kidney Transplantation, Male, Middle Aged, Multivariate Analysis, Rabbits, Renal Insufficiency, Retrospective Studies, Steroids, Time Factors
Show Abstract · Added May 22, 2014
BACKGROUND - Modern immunosuppression and rabbit antithymocyte globulin (rATG) have facilitated the success of early steroid withdrawal (ESW) protocols. Little data exist on optimal rATG dosing in ESW protocols.
METHODS - Rejection at 12 months in era 1 (four doses of rATG, 1.25 mg/kg) vs. era 2 (three doses of rATG, 1.25 mg/kg) was the primary endpoint. Secondary endpoints included patient and graft survival, renal function and infectious complications. Factors associated with rejection at 1 year were identified.
RESULTS - 199 patients received rATG induction and ESW: 102 in era 1 and 97 in era 2. Compared to era 1, era 2 was not associated with worse outcomes, including rejection, renal function, infection or graft survival. Rejection at 1 year and uncensored graft survival differed between the dosing groups. Rejection rates were significantly higher in the <4 mg/kg group compared to the 4-5.9-mg/kg and the ≥6-mg/kg groups, whereas uncensored graft survival was the lowest in the ≥6-mg/kg group. Factors associated with rejection at 12 months included: rATG dose received of 4-5.9 versus <4 mg/kg (OR 0.20, 95% CI 0.036-0.85, p = 0.026); recipient age (per year, OR 0.94, 95% CI 0.89-1.0, p = 0.038); panel reactive antibody 10-79.9 versus <10% (OR 5.4, 95% CI 1.2-25, p = 0.030) and rATG dose held (OR 4.0, 95% CI 1.0-15, p = 0.049).
CONCLUSIONS - A comparison of rATG dosing based on era did not result in a significant difference in rejection, renal function, infection or graft survival. However, when evaluating the study population based on actual dose received there were notable differences in both rejection rates and uncensored graft survival.
© 2013 S. Karger AG, Basel.
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19 MeSH Terms
Less could be better: the case for reducing the dose of cyclophosphamide for children undergoing allogeneic stem cell transplant for severe aplastic anemia.
Hussein AA, Frangoul H
(2013) Pediatr Transplant 17: 326-7
MeSH Terms: Anemia, Aplastic, Antilymphocyte Serum, Cyclophosphamide, Female, Hematopoietic Stem Cell Transplantation, Humans, Immunosuppressive Agents, Male, Transplantation Conditioning, Vidarabine
Added March 7, 2014
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10 MeSH Terms
Risk adopted allogeneic hematopoietic stem cell transplantation using a reduced intensity regimen for children with thalassemia major.
Hussein AA, Al-Zaben A, Ghatasheh L, Natsheh A, Hammada T, Abdel-Rahman F, Abu-Jazar H, Sharma S, Najjar R, Frangoul H
(2013) Pediatr Blood Cancer 60: 1345-9
MeSH Terms: Adolescent, Adult, Antilymphocyte Serum, Busulfan, Child, Child, Preschool, Cyclophosphamide, Disease-Free Survival, Female, Follow-Up Studies, Graft Rejection, Graft Survival, Hematopoietic Stem Cell Transplantation, Humans, Immunologic Factors, Infant, Male, Myeloablative Agonists, Retrospective Studies, Risk Factors, Survival Rate, Tissue Donors, Transplantation Conditioning, Transplantation, Homologous, Vidarabine, beta-Thalassemia
Show Abstract · Added March 7, 2014
BACKGROUND - Patients with thalassemia in developing countries have limited access to safe transfusions, regular medical care and chelation therapy. Although allogeneic hematopoietic stem cell transplantation (HSCT) can offer a curative approach, there are limited data on the use of this procedure in developing countries.
PROCEDURE - Forty-four patients underwent a risk adopted HSCT from matched related family donor in Jordan. Thirty-one patients (7 Class 1 and 24 Class 2) underwent myeloablative conditioning (MAC) with busulfan (16 mg/kg), cyclophosphamide (200 mg/kg) and antithymocyte globulin (ATG). Thirteen patients all with Class 3, seven with hepatitis C received reduced intensity conditioning (RIC) with busulfan (8 mg/kg), fludarabine (175 mg/m(2)), total lymphoid irradiation (500 cGy) and ATG.
RESULTS - All patients had initial neutrophil and platelet engraftment. Secondary graft failure was observed in 2 (6%) patients receiving myeloablative HSCT and 3 (23%) patients receiving RIC. At a median follow up of 64 months (13-108), 43 of 44 patients are alive. The 5-year probability of overall survival (OS) was 97.8% for all patients, 96.8% for patients received MAC and 100% for patients received RIC. The 5-year probability of thalassemia-free survival was 86.4% for all patients, 90.3% and 77% for patients who received MAC and RIC, respectively.
CONCLUSION - Implementing a risk-adopted therapy in patient with thalassemia in Jordan can result in an excellent thalassemia free and OS, especially in those at highest risk.
Copyright © 2013 Wiley Periodicals, Inc.
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26 MeSH Terms
Outcomes with conversion from calcineurin inhibitors to sirolimus after renal transplantation in the context of steroid withdrawal or steroid continuation.
Egbuna OI, Davis RB, Chudinski R, Pavlakis M, Rogers C, Molakatalla P, Johnson SR, Karp S, Monaco AP, Tang H, Hanto DW, Mandelbrot DA
(2009) Transplantation 88: 684-92
MeSH Terms: Adolescent, Adult, Aged, Antilymphocyte Serum, Calcineurin Inhibitors, Child, Child, Preschool, Female, Humans, Immunosuppressive Agents, Infant, Infant, Newborn, Kidney Transplantation, Male, Middle Aged, Mycophenolic Acid, Sirolimus, Steroids, Treatment Outcome
Show Abstract · Added May 22, 2014
BACKGROUND - A number of studies have suggested that conversion from calcineurin inhibitors (CNI) to sirolimus (SRL) can improve graft function in renal transplant patients. None of these studies has converted patients to SRL in the absence of steroids.
METHODS - We describe our experience with 278 renal transplants of which 153 were converted from CNI to SRL. The majority of patients had steroids withdrawn after 6 days. Almost all patients received antithymocyte globulin induction and were maintained on mycophenolate mofetil.
RESULTS - Six months after conversion, patients remaining on SRL therapy had a mean increase in estimated glomerular filtration rate of 6.93 mL/min/1.73 m2 (P<0.0001) compared with preconversion values. SRL-converted patients analyzed by intention-to-treat increased estimated glomerular filtration rate by 5.00 mL/min/1.73 m2 (P=0.0005). Eighty-one percent of patients remaining on SRL had a successful conversion, defined as stable or improved renal function at 6 months. The only factor predictive of unsuccessful conversion was urine protein-to-creatinine ratio more than 1. The benefits of SRL conversion were seen in patients at high immunological risk as well as those at lower risk. Proteinuria increased by a mean of 0.1 (P=0.43) at 6 months. Thirty-six percent of SRL-converted patients experienced adverse effects requiring conversion back to CNI. Rates of rejection, graft loss, and patient death with SRL conversion were low.
CONCLUSIONS - The results from our clinical practice suggest that even in the absence of steroids, SRL conversion significantly improves renal function, with acceptable rates of adverse events.
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19 MeSH Terms
High-dose immunosuppressive therapy and autologous hematopoietic cell transplantation for severe systemic sclerosis: long-term follow-up of the US multicenter pilot study.
Nash RA, McSweeney PA, Crofford LJ, Abidi M, Chen CS, Godwin JD, Gooley TA, Holmberg L, Henstorf G, LeMaistre CF, Mayes MD, McDonagh KT, McLaughlin B, Molitor JA, Nelson JL, Shulman H, Storb R, Viganego F, Wener MH, Seibold JR, Sullivan KM, Furst DE
(2007) Blood 110: 1388-96
MeSH Terms: Adult, Antigens, CD34, Antilymphocyte Serum, Combined Modality Therapy, Cyclophosphamide, Female, Fibrosis, Follow-Up Studies, Granulocyte Colony-Stimulating Factor, Hematopoietic Stem Cell Mobilization, Hematopoietic Stem Cell Transplantation, Humans, Immunosuppressive Agents, Male, Middle Aged, Pilot Projects, Scleroderma, Systemic, Skin, Survival Rate, Transplantation, Autologous, Whole-Body Irradiation
Show Abstract · Added September 18, 2013
More effective therapeutic strategies are required for patients with poor-prognosis systemic sclerosis (SSc). A phase 2 single-arm study of high-dose immunosuppressive therapy (HDIT) and autologous CD34-selected hematopoietic cell transplantation (HCT) was conducted in 34 patients with diffuse cutaneous SSc. HDIT included total body irradiation (800 cGy) with lung shielding, cyclophosphamide (120 mg/kg), and equine antithymocyte globulin (90 mg/kg). Neutrophil and platelet counts were recovered by 9 (range, 7 to 13) and 11 (range, 7 to 25) days after HCT, respectively. Seventeen of 27 (63%) evaluable patients who survived at least 1 year after HDIT had sustained responses at a median follow-up of 4 (range, 1 to 8) years. There was a major improvement in skin (modified Rodnan skin score, -22.08; P < .001) and overall function (modified Health Assessment Questionnaire Disability Index, -1.03; P < .001) at final evaluation. Importantly, for the first time, biopsies confirmed a statistically significant decrease of dermal fibrosis compared with baseline (P < .001). Lung, heart, and kidney function, in general, remained clinically stable. There were 12 deaths during the study (transplantation-related, 8; SSc-related, 4). The estimated progression-free survival was 64% at 5 years. Sustained responses including a decrease in dermal fibrosis were observed exceeding those previously reported with other therapies. HDIT and autologous HCT for SSc should be evaluated in a randomized clinical trial.
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21 MeSH Terms
Serum sickness following rabbit antithymocyte-globulin induction in a liver transplant recipient: case report and literature review.
Lundquist AL, Chari RS, Wood JH, Miller GG, Schaefer HM, Raiford DS, Wright KJ, Gorden DL
(2007) Liver Transpl 13: 647-50
MeSH Terms: Acute Kidney Injury, Animals, Antibodies, Monoclonal, Antilymphocyte Serum, Female, Glucocorticoids, Humans, Injections, Intravenous, Liver Transplantation, Methylprednisolone, Middle Aged, Rabbits, Serum Sickness, Treatment Outcome
Show Abstract · Added February 25, 2014
Thymoglobulin (Genzyme, Cambridge, MA) is an antithymocyte globulin preparation used for induction immunosuppression therapy in solid organ transplantation. It is being utilized with increasing frequency in orthotopic liver transplantation (OLT) in an effort to minimize or delay the use of calcineurin inhibitors due to their inherent nephrotoxicity. Experience with thymoglobulin in OLT remains limited. We report a case of serum sickness in a patient who received thymoglobulin following OLT. The patient experienced intermittent fevers, polyarthralgia, and acute renal failure 9 days after completion of thymoglobulin administration. The patient's symptoms resolved rapidly and completely with a course of intravenous steroids. We review a set of diagnostic criteria for serum sickness and emphasize the importance of early recognition of the process. Early treatment of serum sickness with steroids or plasmapheresis is highly effective and can reduce unnecessary morbidity from this unusual sequela of induction immunosuppression with antithymocyte globulin.
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14 MeSH Terms
Busulfan/melphalan/antithymocyte globulin followed by unrelated donor cord blood transplantation for treatment of infant leukemia and leukemia in young children: the Cord Blood Transplantation study (COBLT) experience.
Wall DA, Carter SL, Kernan NA, Kapoor N, Kamani NR, Brochstein JA, Frangoul H, Goyal RK, Horan JT, Pietryga D, Wagner JE, Kurtzberg J, COBLT Steering Committee
(2005) Biol Blood Marrow Transplant 11: 637-46
MeSH Terms: Antilymphocyte Serum, Antineoplastic Agents, Alkylating, Antineoplastic Combined Chemotherapy Protocols, Busulfan, Child, Preschool, Cord Blood Stem Cell Transplantation, Disease-Free Survival, Female, Graft Survival, Graft vs Host Disease, Histocompatibility Testing, Humans, Immunosuppressive Agents, Infant, Infant, Newborn, Leukemia, Male, Melphalan, Retrospective Studies, Transplantation Conditioning, Whole-Body Irradiation
Show Abstract · Added March 27, 2014
A non-total body irradiation-containing preparative regimen was studied in young children (<4 years old) undergoing unrelated donor cord blood transplantation as part of the Cord Blood Transplantation trial for the treatment of acute lymphoblastic leukemia (n = 14), acute myeloid leukemia (n = 13), undifferentiated leukemia (n = 1), juvenile myelomonocytic leukemia (n = 2), and myelodysplastic syndromes (n = 2). Donor/recipient HLA matching based on low-/intermediate-resolution molecular typing for HLA-A and -B and high-resolution HLA-DRB1 typing was 5/6 or 6/6 (n = 21) or 4/6 (n = 11). The preparative therapy consisted of busulfan, melphalan, and antithymocyte globulin, with cyclosporine and corticosteroids for graft-versus-host disease (GVHD) prophylaxis. The median age was 1.6 years (range, 0.5-3.9 years), and the median weight was 10.5 kg (range, 5.8-19.5 kg). Cord blood grafts contained a median of 10.7 x 10 7 nucleated cells per kilogram (range, 4.6-29.2) and 2.6 x 10(5) CD34+ cells per kilogram (range, 0.7-8.3). The cumulative incidence (CINC) of neutrophil recovery (absolute neutrophil count >500/microL) at day 42 was 0.59 (95% confidence interval [CI], 0.44-0.78) at a median of 31 days (range, 23-55 days). The CINC and Kaplan-Meier estimates of platelet engraftment at day 180 were 0.53 (95% CI, 0.34-0.69) and 0.82 (95% CI, 0.61-1.00), respectively. CINC estimates of grade III/IV acute GVHD at day 100 and chronic GVHD at 1 year were 0.25 (95% CI, 0.09-0.41) and 0.26 (95% CI, 0.09-0.44), respectively. The CINC estimate of relapse was 0.31 (95% CI, 0.16-0.47) at 2 years. With a median follow-up of 27.8 months (range, 23.4-46.7 months), the probability of survival at 1 year was 0.47 (95% CI, 0.30-0.64). A preparative regimen containing a busulfan/melphalan/antithymocyte globulin preparative regimen is well tolerated in the setting of unrelated donor cord blood transplantation for childhood leukemia and can serve as a platform preparative regimen for intensifying host immunosuppression and antileukemic therapy to allow for improved engraftment and improved relapse-free survival.
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21 MeSH Terms
Polyclonal antibody-induced serum sickness in renal transplant recipients: treatment with therapeutic plasma exchange.
Tanriover B, Chuang P, Fishbach B, Helderman JH, Kizilisik T, Nylander W, Shaffer D, Langone AJ
(2005) Transplantation 80: 279-81
MeSH Terms: Adult, Animals, Antilymphocyte Serum, Female, Horses, Humans, Immunosuppressive Agents, Kidney Transplantation, Male, Mice, Middle Aged, Plasma Exchange, Rabbits, Serum Sickness, Tissue Donors
Show Abstract · Added March 4, 2014
Serum sickness is an immune-complex mediated illness that frequently occurs in patients after polyclonal antibody therapy (ATGAM or thymoglobulin). Serum sickness presents with significant morbidity but is self-limited and resolves with prolonged steroid therapy. We present five renal transplant patients who developed serum sickness after polyclonal antibody treatment with severe symptoms that persisted after being started on systemic steroids. These patients underwent one or two courses of therapeutic plasma exchange (TPE) with subsequent complete resolution of their symptoms. Renal transplant recipients with serum sickness after polyclonal antibody therapy may benefit from TPE by accelerating their time to recovery and thereby reducing overall morbidity.
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15 MeSH Terms