Durable Chimerism and Long-Term Survival after Unrelated Umbilical Cord Blood Transplantation for Pediatric Hemophagocytic Lymphohistiocytosis: A Single-Center Experience

Biology of Blood and Marrow Transplantation

Volumn 23, Issue 10, 2017, Pages 1722-1728

  Patel, Sachit A ^a   Allewelt, Heather A ^b   Troy, Jesse D ^c   Martin, Paul L ^d   Driscoll, Timothy A ^d   Prasad, Vinod K ^d   Kurtzberg, Joanne ^c,d   Page, Kristin M ^d   Parikh, Suhag H ^d  

^a UNIVERSITY OF NEBRASKA MEDICAL CENTER   (United States)

^b FLORIDA HOSPITAL   (United States)

^c DUKE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^d DUKE UNIVERSITY MEDICAL CENTER   (United States)

Author keywords

Hemophagocytic lymphohistiocytosis; HLH; Pediatric; Stem cell transplantation; Umbilical cord blood transplantation

Indexed keywords

ACICLOVIR; ALEMTUZUMAB; AMPHOTERICIN B; BUSULFAN; CYCLOPHOSPHAMIDE; CYCLOSPORIN; ETOPOSIDE; FILGRASTIM; FLUDARABINE; HEPARIN; HYDROXYUREA; IMMUNOGLOBULIN; LEVETIRACETAM; MELPHALAN; MESNA; METHYLPREDNISOLONE; MYCOPHENOLATE MOFETIL; PHENYTOIN; TACROLIMUS; THIOTEPA; THYMOCYTE ANTIBODY; URSODEOXYCHOLIC ACID; VORICONAZOLE;

ACUTE GRAFT VERSUS HOST DISEASE; ACUTE KIDNEY FAILURE; ADENOVIRUS INFECTION; ADOLESCENT; ADULT RESPIRATORY DISTRESS SYNDROME; ARTICLE; AUTOIMMUNE THYROIDITIS; BACTEREMIA; CATARACT; CELL TRANSPLANTATION; CHILD; CHIMERA; CHRONIC GRAFT VERSUS HOST DISEASE; CLINICAL ARTICLE; CLINICAL EFFECTIVENESS; COLITIS; CONFIDENCE INTERVAL; CONTINUOUS INFUSION; DISEASE FREE SURVIVAL; DONOR; ENGRAFTMENT; EVENT FREE SURVIVAL; FEMALE; GRAFT FAILURE; GRANULOCYTE; HEMOPHAGOCYTIC SYNDROME; HUMAN; INCIDENCE; INVASIVE CANDIDIASIS; LONG TERM SURVIVAL; LOW DRUG DOSE; MALE; MULTIPLE ORGAN FAILURE; NEUROLOGIC DISEASE; NEUTROPHIL; OSTEOCHONDROMA; OUTCOME ASSESSMENT; PARAINFLUENZA VIRUS INFECTION; POSTERIOR REVERSIBLE ENCEPHALOPATHY SYNDROME; PRESCHOOL CHILD; SCHOOL CHILD; SEIZURE; SEPSIS; TRANSPLANTATION; TRANSPLANTATION CONDITIONING; TREATMENT OUTCOME; UMBILICAL CORD BLOOD; UMBILICAL CORD BLOOD TRANSPLANTATION; VEIN OCCLUSION; COMPLICATION; CORD BLOOD STEM CELL TRANSPLANTATION; CYTOLOGY; FETUS BLOOD; GRAFT SURVIVAL; GRAFT VERSUS HOST REACTION; INFANT; MORTALITY; PROCEDURES; STANDARDS;

ADOLESCENT; CHILD; CHILD, PRESCHOOL; CHIMERISM; CORD BLOOD STEM CELL TRANSPLANTATION; DISEASE-FREE SURVIVAL; FETAL BLOOD; GRAFT SURVIVAL; GRAFT VS HOST DISEASE; HUMANS; INFANT; LYMPHOHISTIOCYTOSIS, HEMOPHAGOCYTIC; TRANSPLANTATION CONDITIONING;

EID: 85025470329     PISSN: 10838791     EISSN: 15236536     Source Type: Journal    
DOI: 10.1016/j.bbmt.2017.06.013     Document Type: Article

References (37)

1. Janka, G.E., Lehmberg, K., Hemophagocytic syndromes–an update. Blood Rev 28 (2014), 135–142.
2. Chandrakasan, S., Filipovich, A.H., Hemophagocytic lymphohistiocytosis: advances in pathophysiology, diagnosis, and treatment. J Pediatr 163 (2013), 1253–1259.
3. Seo, J.J., Hematopoietic cell transplantation for hemophagocytic lymphohistiocytosis: recent advances and controversies. Blood Res 50 (2015), 131–139.
4. Henter, J.I., Horne, A., Arico, M., et al. HLH-2004: diagnostic and therapeutic guidelines for hemophagocytic lymphohistiocytosis. Pediatr Blood Cancer 48 (2007), 124–131.
5. Stepp, S.E., Dufourcq-Lagelouse, R., Le Deist, F., et al. Perforin gene defects in familial hemophagocytic lymphohistiocytosis. Science 286 (1999), 1957–1959.
6. Janka, G., Familial and acquired hemophagocytic lymphohistiocytosis. Annu Rev Med 63 (2012), 233–246.
7. zur Stadt, U., Schmidt, S., Kasper, B., et al. Linkage of familial hemophagocytic lymphohistiocytosis (FHL) type-4 to chromosome 6q24 and identification of mutations in syntaxin 11. Hum Mol Genet 14 (2005), 827–834.
8. Feldmann, J., Callebaut, I., Raposo, G., et al. Munc13-4 is essential for cytolytic granules fusion and is mutated in a form of familial hemophagocytic lymphohistiocytosis (FHL3). Cell 115 (2003), 461–473.
9. Jordan, M.B., Allen, C.E., Weitzman, S., Filipovich, A.H., McClain, K.L., How I treat hemophagocytic lymphohistiocytosis. Blood 118 (2011), 4041–4052.
10. Baker, K.S., Filipovich, A.H., Gross, T.G., et al. Unrelated donor hematopoietic cell transplantation for hemophagocytic lymphohistiocytosis. Bone Marrow Transplant 42 (2008), 175–180.
11. Cesaro, S., Locatelli, F., Lanino, E., et al. Hematopoietic stem cell transplantation for hemophagocytic lymphohistiocytosis: a retrospective analysis of data from the Italian Association of Pediatric Hematology Oncology (AIEOP). Haematologica 93 (2008), 1694–1701.
12. Ouachée-Chardin, M., Elie, C., de Saint Basile, G., et al. Hematopoietic stem cell transplantation in hemophagocytic lymphohistiocytosis: a single-center report of 48 patients. Pediatrics 117 (2006), e743–e750.
13. Horne, A., Janka, G., Maarten Egeler, R., et al. Haematopoietic stem cell transplantation in haemophagocytic lymphohistiocytosis. Br J Haematol 129 (2005), 622–630.
14. Trottestam, H., Horne, A., Aricò, M., et al. Chemoimmunotherapy for hemophagocytic lymphohistiocytosis: long-term results of the HLH-94 treatment protocol. Blood 118 (2011), 4577–4584.
15. Cooper, N., Rao, K., Goulden, N., Webb, D., Amroli, A.P., Veys, P., The use of reduced-intensity stem cell transplantation in haemophagocytic lymphohistiocytosis and Langerhans cell histiocytosis. Bone Marrow Transplant 42 (2008), S47–S50.
16. Marsh, R.A., Kim, M.-O., Liu, C., et al. An intermediate alemtuzumab schedule reduces the incidence of mixed chimerism following reduced-intensity conditioning hematopoietic cell transplantation for hemophagocytic lymphohistiocytosis. Biol Blood Marrow Transplant 19 (2013), 1625–1631.
17. Marsh, R.A., Vaughn, G., Kim, M.-O., et al. Reduced-intensity conditioning significantly improves survival of patients with hemophagocytic lymphohistiocytosis undergoing allogeneic hematopoietic cell transplantation. Blood 116 (2010), 5824–5831.
18. Lehmberg, K., Albert, M.H., Beier, R., et al. Treosulfan-based conditioning regimen for children and adolescents with hemophagocytic lymphohistiocytosis. Haematologica 99 (2014), 180–184.
19. Nishi, M., Nishimura, R., Suzuki, N., et al. Reduced-intensity conditioning in unrelated donor cord blood transplantation for familial hemophagocytic lymphohistiocytosis. Am J Hematol 87 (2012), 637–639.
20. Sawada, A., Ohga, S., Ishii, E., et al. Feasibility of reduced-intensity conditioning followed by unrelated cord blood transplantation for primary hemophagocytic lymphohistiocytosis: a nationwide retrospective analysis in Japan. Int J Hematol 98 (2013), 223–230.
21. Frangoul, H., Wang, L., Harrell, F.E. Jr, Manes, B., Calder, C., Domm, J., Unrelated umbilical cord blood transplantation in children with immune deficiency: results of a multicenter study. Bone Marrow Transplant 45 (2010), 283–288.
22. Hinson, A., Owen, W., Prose, N., Parikh, S., Thornburg, C., Congenital hemophagocytic lymphohistiocytosis presenting as thrombocytopenia in a newborn. J Pediatr Hematol Oncol 37 (2015), 300–303.
23. Parikh, S.H., Mendizabal, A., Benjamin, C.L., et al. A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases. Biol Blood Marrow Transplant 20 (2014), 326–336.
24. Wall, D.A., Carter, S.L., Kernan, N.A., et al. 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. Biol Blood Marrow Transplant 11 (2005), 637–646.
25. Prasad, V.K., Mendizabal, A., Parikh, S.H., et al. Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes. Blood 112 (2008), 2979–2989.
26. Rubinstein, P., Dobrila, L., Rosenfield, R.E., et al. Processing and cryopreservation of placental/umbilical cord blood for unrelated bone marrow reconstitution. Proc Natl Acad Sci USA 92 (1995), 10119–10122.
27. Przepiorka, D., Weisdorf, D., Martin, P., et al. 1994 consensus conference on acute GVHD grading. Bone Marrow Transplant 15 (1995), 825–828.
28. Jagasia, M.H., Greinix, H.T., Arora, M., et al. National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. The 2014 diagnosis and staging working group report. Biol Blood Marrow Transplant 21 (2015), 389–401 e381.
29. Ohga, S., Kudo, K., Ishii, E., et al. Hematopoietic stem cell transplantation for familial hemophagocytic lymphohistiocytosis and Epstein-Barr virus–associated hemophagocytic lymphohistiocytosis in Japan. Pediatr Blood Cancer 54 (2010), 299–306.
30. Yoon, H.S., Im, H.J., Moon, H.N., et al. The outcome of hematopoietic stem cell transplantation in Korean children with hemophagocytic lymphohistiocytosis. Pediatr Transplant 14 (2010), 735–740.
31. Page, K.M., Zhang, L., Mendizabal, A., et al. Total colony-forming units are a strong, independent predictor of neutrophil and platelet engraftment after unrelated umbilical cord blood transplantation: a single-center analysis of 435 cord blood transplants. Biol Blood Marrow Transplant 17 (2011), 1362–1374.
32. Eapen, M., Ahn, K.W., Orchard, P.J., et al. Long-term survival and late deaths after hematopoietic cell transplantation for primary immunodeficiency diseases and inborn errors of metabolism. Biol Blood Marrow Transplant 18 (2012), 1438–1445.
33. Hartz, B., Marsh, R., Rao, K., et al. The minimum required level of donor chimerism in hereditary hemophagocytic lymphohistiocytosis. Blood 127 (2016), 3281–3290.
34. Terrell, C.E., Jordan, M.B., Mixed hematopoietic or T-cell chimerism above a minimal threshold restores perforin-dependent immune regulation in perforin-deficient mice. Blood 122 (2013), 2618–2621.
35. Cooper, N., Rao, K., Gilmour, K., et al. Stem cell transplantation with reduced-intensity conditioning for hemophagocytic lymphohistiocytosis. Blood 107 (2006), 1233–1236.
36. Marsh, R., Ellen, C., McClain, K., et al. Salvage therapy of refractory hemophagocytic lymphohistiocytosis with alemtuzumab. Pediatr Blood Cancer 60 (2013), 101–109.
37. Kamani, N.R., Walters, M.C., Carter, S., et al. Unrelated donor cord blood transplantation for children with severe sickle cell disease: results of one cohort from the phase II study from the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). Biol Blood Marrow Transplant 18 (2012), 1265–1272.