Academic Appointments


Clinical Trials


  • Stem Cell Transplant From Donors After Alpha Beta Cell Depletion in Children and Young Adults Recruiting

    The purpose of the CliniMACS® TCRαβ-Biotin System and CliniMACS® CD19 is to improve the safety and efficacy of allogeneic HLA-partially matched related or unrelated donors HSCT when no matched donors are available, to treat malignant and nonmalignant disorders for which HSCT is the recommended best available therapy. Initially this device will be used in a single-center, open-label, single-arm, phase II clinical trial to evaluate the efficacy of haploidentical PBSC grafts depleted of TCRα/β+ and CD19+ cells using the CliniMACS® TCRαβ/CD19 System in children and adults with hematological and non-hematological malignancies.

    View full details

All Publications


  • Experience with Ruxolitinib (Jakafi (R)) As a Salvage Therapy for Graft-Versus-Host Disease in Children and Young Adults Mavers, M., Klinger, E., Shyr, D. C., Shah, A. J., Bertaina, A., Soni, S. ELSEVIER SCIENCE INC. 2020: S179
  • Transplantation Outcomes for Children with Severe Combined Immune Deficiency (SCID) Have Improved over Time: A 36-Year Summary Report By the Primary Immune Deficiency Treatment Consortium (PIDTC) Thakar, M. S., Logan, B., Buckley, R. H., Haddad, E., Dvorak, C. C., O'Reilly, R. J., Kapoor, N., Satter, L., Martinez, C., Pai, S., Heimall, J., Jyonouchi, S., Sullivan, K. E., Chandra, S., Smith, A. R., Chaudhury, S., Saldana, B., Sunkersett, G., Shyr, D. C., Burroughs, L. M., Petrovic, A., Quigg, T. C., Shenoy, S., Bednarski, J. J., DeSantes, K., Cuvelier, G. E., Chandrakasan, S., Gillio, A. P., Knutsen, A. P., Eissa, H., Goldman, F., Moore, T. B., Aquino, V., Shereck, E., Lugt, M., Caywood, E. H., Yu, L. C., Rozmus, J., Talano, J. M., Malech, H. L., Shah, A. J., Abu-Arja, R., Miller, H. K., Bani-Hashemi, T., Chang, C. K., Dunn, E., Torgerson, T., Pulsipher, M. A., Griffith, L. M., Cowan, M. J., Kohn, D. B., Puck, J., Notarangelo, L. D. ELSEVIER SCIENCE INC. 2020: S18–S19
  • Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey. Frontiers in immunology Chan, A. Y., Leiding, J. W., Liu, X., Logan, B. R., Burroughs, L. M., Allenspach, E. J., Skoda-Smith, S., Uzel, G., Notarangelo, L. D., Slatter, M., Gennery, A. R., Smith, A. R., Pai, S., Jordan, M. B., Marsh, R. A., Cowan, M. J., Dvorak, C. C., Craddock, J. A., Prockop, S. E., Chandrakasan, S., Kapoor, N., Buckley, R. H., Parikh, S., Chellapandian, D., Oshrine, B. R., Bednarski, J. J., Cooper, M. A., Shenoy, S., Davila Saldana, B. J., Forbes, L. R., Martinez, C., Haddad, E., Shyr, D. C., Chen, K., Sullivan, K. E., Heimall, J., Wright, N., Bhatia, M., Cuvelier, G. D., Goldman, F. D., Meyts, I., Miller, H. K., Seidel, M. G., Vander Lugt, M. T., Bacchetta, R., Weinacht, K. G., Andolina, J. R., Caywood, E., Chong, H., de la Morena, M. T., Aquino, V. M., Shereck, E., Walter, J. E., Dorsey, M. J., Seroogy, C. M., Griffith, L. M., Kohn, D. B., Puck, J. M., Pulsipher, M. A., Torgerson, T. R. 2020; 11: 239

    Abstract

    Primary Immune Regulatory Disorders (PIRD) are an expanding group of diseases caused by gene defects in several different immune pathways, such as regulatory T cell function. Patients with PIRD develop clinical manifestations associated with diminished and exaggerated immune responses. Management of these patients is complicated; oftentimes immunosuppressive therapies are insufficient, and patients may require hematopoietic cell transplant (HCT) for treatment. Analysis of HCT data in PIRD patients have previously focused on a single gene defect. This study surveyed transplanted patients with a phenotypic clinical picture consistent with PIRD treated in 33 Primary Immune Deficiency Treatment Consortium centers and European centers. Our data showed that PIRD patients often had immunodeficient and autoimmune features affecting multiple organ systems. Transplantation resulted in resolution of disease manifestations in more than half of the patients with an overall 5-years survival of 67%. This study, the first to encompass disorders across the PIRD spectrum, highlights the need for further research in PIRD management.

    View details for DOI 10.3389/fimmu.2020.00239

    View details for PubMedID 32153572

  • SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery BLOOD Haddad, E., Logan, B. R., Griffith, L. M., Buckley, R. H., Parrott, R. E., Prockop, S. E., Small, T. N., Chaisson, J., Dvorak, C. C., Mumane, M., Kapoor, N., Abdel-Azim, H., Hanson, I. C., Martinez, C., Bleesing, J. H., Chandra, S., Smith, A. R., Cavanaugh, M. E., Jyonouchi, S., Sullivan, K. E., Burroughs, L., Skoda-Smith, S., Haight, A. E., Tumlin, A. G., Quigg, T. C., Taylor, C., Saldana, B., Keller, M. D., Seroogy, C. M., Desantes, K. B., Petrovic, A., Leiding, J. W., Shyr, D. C., Decaluwe, H., Teira, P., Gillio, A. P., Knutsen, A. P., Moore, T. B., Kletzel, M., Craddock, J. A., Aquino, V., Davis, J. H., Yu, L. C., Cuvelier, G. E., Bednarski, J. J., Goldman, F. D., Kang, E. M., Shereck, E., Porteus, M. H., Connelly, J. A., Fleisher, T. A., Malech, H. L., Shearer, W. T., Szabolcs, P., Thakar, M. S., Vander Lugt, M. T., Heimall, J., Yin, Z., Pulsipher, M. A., Pai, S., Kohn, D. B., Puck, J. M., Cowan, M. J., O'Reilly, R. J., Notarangelo, L. D. 2018; 132 (17): 1737–49

    Abstract

    The Primary Immune Deficiency Treatment Consortium (PIDTC) performed a retrospective analysis of 662 patients with severe combined immunodeficiency (SCID) who received a hematopoietic cell transplantation (HCT) as first-line treatment between 1982 and 2012 in 33 North American institutions. Overall survival was higher after HCT from matched-sibling donors (MSDs). Among recipients of non-MSD HCT, multivariate analysis showed that the SCID genotype strongly influenced survival and immune reconstitution. Overall survival was similar for patients with RAG, IL2RG, or JAK3 defects and was significantly better compared with patients with ADA or DCLRE1C mutations. Patients with RAG or DCLRE1C mutations had poorer immune reconstitution than other genotypes. Although survival did not correlate with the type of conditioning regimen, recipients of reduced-intensity or myeloablative conditioning had a lower incidence of treatment failure and better T- and B-cell reconstitution, but a higher risk for graft-versus-host disease, compared with those receiving no conditioning or immunosuppression only. Infection-free status and younger age at HCT were associated with improved survival. Typical SCID, leaky SCID, and Omenn syndrome had similar outcomes. Landmark analysis identified CD4+ and CD4+CD45RA+ cell counts at 6 and 12 months post-HCT as biomarkers predictive of overall survival and long-term T-cell reconstitution. Our data emphasize the need for patient-tailored treatment strategies depending upon the underlying SCID genotype. The prognostic significance of CD4+ cell counts as early as 6 months after HCT emphasizes the importance of close follow-up of immune reconstitution to identify patients who may need additional intervention to prevent poor long-term outcome.

    View details for PubMedID 30154114

    View details for PubMedCentralID PMC6202916