Stem Cell Transplant for Immunologic or Histiocytic Disorders
Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders, Chediak-Higashi Syndrome
About this trial
This is an interventional treatment trial for Hemophagocytic Lymphohistiocytosis focused on measuring Stem cell transplant, immunodeficiency, donor lymphocyte infusion
Eligibility Criteria
Inclusion Criteria: Patients with immunodeficiencies or histiocytic disorders 0-35 years of age with an acceptable stem cell donor and disease characteristic defined by the following: Patients with histocytic disorders (hemophagocytic lymphohistiocytosis of any etiology and refractory Langerhans cell histiocytosis) who do not meet eligibility criteria for a myeloablative transplant procedure Patients with immunodeficiency disorders in whom residual immune function may not require a fully myeloablative preparative regimen or patient is ineligible for standard myeloablative preparative regimen (any form of severe combined immunodeficiency [SCID], or other immunodeficiency with T cell defect) Patients with immunodeficiency disorders that have had poor outcome with myeloablative stem cell transplants (including, but not limited to, common variable immunodeficiency [CVID], Wiskott Aldrich Syndrome [WAS] if > 5 years of age, ataxia telangiectasia) Patients with immunodeficiencies or histocytic disorders that require a second stem cell transplant (SCT) for any reason Exclusion Criteria: Karnofsky or Lansky performance score <70 Glomerular filtration rate (GFR)<30% predicted Cardiac function <50% normal by echocardiogram Serum creatinine > 2x normal for age/weight Pregnant or lactating females Active serious infection that has not had an adequate course of therapy pre-SCT. Any patient with acquired immune deficiency syndrome (AIDS) or AIDS-related complex (ARC) or human immunodeficiency virus (HIV) seropositivity
Sites / Locations
- Masonic Cancer Center University of Minnesota
Arms of the Study
Arm 1
Arm 2
Arm 3
Active Comparator
Active Comparator
Active Comparator
Arm 1 - Matched sibling donor
Arm 2 - Matched unrelated donor
Arm 3 - Mismatched double cord donors
Stem Cell Transplant: human leukocyte antigen (HLA) genotypic matched sibling donor and pre-treatment with fludarabine, melphalan, anti-thymocyte globulin or Campath 1H and post-treatment with Cyclosporin A, mycophenolate mofetil and Intravenous immunoglobulin (IVIG)
Stem Cell Transplant: HLA phenotypic matched unrelated peripheral blood stem cell (PBSC) donor and pre-treatment with fludarabine, melphalan, anti-thymocyte globulin or Campath 1H and post-treatment with Cyclosporin A, mycophenolate mofetil and Intravenous immunoglobulin (IVIG)
Stem Cell Transplant: two HLA 0-2 antigen mismatched unrelated cord blood donors (double cord) and pre-treatment with fludarabine, melphalan, anti-thymocyte globulin or Campath 1H and post-treatment with Cyclosporin A, mycophenolate mofetil and Intravenous immunoglobulin (IVIG)