Literature DB >> 26453586

Haploidentical Hematopoietic Cell Transplantation with Post-Transplant Cyclophosphamide in a Patient with Chronic Granulomatous Disease and Active Infection: A First Report.

Mark Parta1, Dianne Hilligoss2, Corin Kelly2, Nana Kwatemaa2, Narda Theobald2, Harry Malech2, Elizabeth M Kang2.   

Abstract

PURPOSE: We describe haploidentical hematopoietic cell transplantation (HCT) with high-dose post-transplant cyclophosphamide (PTCy) in a boy with x-linked chronic granulomatous disease (CGD).
METHODS: A persistent and life-threatening fungal infection was the indication for HSCT. Non-myeloablative conditioning with PTCy (50 mg/kg days 3 and 4) was used in the absence of fully matched donors.
RESULTS: Engraftment occurred on day 24. The patient experienced Grade 2 graft-versus-host disease of the skin and gastrointestinal tract and CMV infection, both of which were controlled. Chimerism was 100 % at days 30 and 6 months. Cessation of antifungal therapy was consistent with cure of the infection.
CONCLUSIONS: Haploidentical HCT with high-dose PTCy for CGD is feasible and succeeded even in the context of active infection.

Entities:  

Keywords:  Chronic granulomatous disease; graft versus host disease; haploidentical; hematopoietic cell transplantation; post-transplant cyclophosphamide; scedosporium apiospermum

Mesh:

Substances:

Year:  2015        PMID: 26453586      PMCID: PMC6317348          DOI: 10.1007/s10875-015-0204-y

Source DB:  PubMed          Journal:  J Clin Immunol        ISSN: 0271-9142            Impact factor:   8.317


  36 in total

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Authors:  Jonathan D Powell; Courtney Fitzhugh; Elizabeth M Kang; Mathew Hsieh; Ronald H Schwartz; John F Tisdale
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2.  Depletion of naive T cells using clinical grade magnetic CD45RA beads: a new approach for GVHD prophylaxis.

Authors:  D Teschner; E Distler; D Wehler; M Frey; D Marandiuc; K Langeveld; M Theobald; S Thomas; W Herr
Journal:  Bone Marrow Transplant       Date:  2013-08-12       Impact factor: 5.483

3.  Multi-institutional study of post-transplantation cyclophosphamide as single-agent graft-versus-host disease prophylaxis after allogeneic bone marrow transplantation using myeloablative busulfan and fludarabine conditioning.

Authors:  Christopher G Kanakry; Paul V O'Donnell; Terry Furlong; Marcos J de Lima; Wei Wei; Marta Medeot; Marco Mielcarek; Richard E Champlin; Richard J Jones; Peter F Thall; Borje S Andersson; Leo Luznik
Journal:  J Clin Oncol       Date:  2014-09-29       Impact factor: 44.544

4.  Durable engraftment of major histocompatibility complex-incompatible cells after nonmyeloablative conditioning with fludarabine, low-dose total body irradiation, and posttransplantation cyclophosphamide.

Authors:  L Luznik; S Jalla; L W Engstrom; R Iannone; E J Fuchs
Journal:  Blood       Date:  2001-12-01       Impact factor: 22.113

Review 5.  Haploidentical transplantation for hematologic malignancies: where do we stand?

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6.  Diagnostic and therapeutic impact of whole body positron emission tomography using fluorine-18-fluoro-2-deoxy-D-glucose in children with chronic granulomatous disease.

Authors:  T Güngör; I Engel-Bicik; G Eich; U V Willi; D Nadal; J P Hossle; R A Seger; H C Steinert
Journal:  Arch Dis Child       Date:  2001-10       Impact factor: 3.791

7.  Periostitis secondary to prolonged voriconazole therapy in lung transplant recipients.

Authors:  T F Wang; T Wang; R Altman; P Eshaghian; J P Lynch; D J Ross; J A Belperio; S S Weigt; R Saggar; A Gregson; B Kubak; R Saggar
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8.  Successful treatment of invasive aspergillosis in chronic granulomatous disease by bone marrow transplantation, granulocyte colony-stimulating factor-mobilized granulocytes, and liposomal amphotericin-B.

Authors:  H Ozsahin; M von Planta; I Müller; H C Steinert; D Nadal; R Lauener; P Tuchschmid; U V Willi; M Ozsahin; N E Crompton; R A Seger
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9.  Transplantation outcomes for severe combined immunodeficiency, 2000-2009.

Authors:  Sung-Yun Pai; Brent R Logan; Linda M Griffith; Rebecca H Buckley; Roberta E Parrott; Christopher C Dvorak; Neena Kapoor; Imelda C Hanson; Alexandra H Filipovich; Soma Jyonouchi; Kathleen E Sullivan; Trudy N Small; Lauri Burroughs; Suzanne Skoda-Smith; Ann E Haight; Audrey Grizzle; Michael A Pulsipher; Ka Wah Chan; Ramsay L Fuleihan; Elie Haddad; Brett Loechelt; Victor M Aquino; Alfred Gillio; Jeffrey Davis; Alan Knutsen; Angela R Smith; Theodore B Moore; Marlis L Schroeder; Frederick D Goldman; James A Connelly; Matthew H Porteus; Qun Xiang; William T Shearer; Thomas A Fleisher; Donald B Kohn; Jennifer M Puck; Luigi D Notarangelo; Morton J Cowan; Richard J O'Reilly
Journal:  N Engl J Med       Date:  2014-07-31       Impact factor: 91.245

10.  Human cytomegalovirus replication is strictly inhibited by siRNAs targeting UL54, UL97 or UL122/123 gene transcripts.

Authors:  Stuart T Hamilton; Jens Milbradt; Manfred Marschall; William D Rawlinson
Journal:  PLoS One       Date:  2014-06-02       Impact factor: 3.240

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  15 in total

1.  Alternative-Donor Hematopoietic Stem Cell Transplantation with Post-Transplantation Cyclophosphamide for Nonmalignant Disorders.

Authors:  Orly R Klein; Allen R Chen; Christopher Gamper; David Loeb; Elias Zambidis; Nicolas Llosa; Jeffrey Huo; Amy E Dezern; Diana Steppan; Nancy Robey; Mary Jo Holuba; Kenneth R Cooke; Heather J Symons
Journal:  Biol Blood Marrow Transplant       Date:  2016-02-06       Impact factor: 5.742

Review 2.  Enzymatic Mechanisms Involved in Evasion of Fungi to the Oxidative Stress: Focus on Scedosporium apiospermum.

Authors:  C Staerck; P Vandeputte; A Gastebois; A Calenda; S Giraud; N Papon; J P Bouchara; M J J Fleury
Journal:  Mycopathologia       Date:  2017-06-21       Impact factor: 2.574

3.  Granulocyte transfusions in patients with chronic granulomatous disease and refractory infections: The NIH experience.

Authors:  Beatriz E Marciano; Elisabeth S Allen; Cathy Conry-Cantilena; Ervand Kristosturyan; Harvey G Klein; Thomas A Fleisher; Steven M Holland; Harry L Malech; Sergio D Rosenzweig
Journal:  J Allergy Clin Immunol       Date:  2017-03-22       Impact factor: 10.793

4.  Successful Haploidentical Stem Cell Transplantation with Post-Transplant Cyclophosphamide in a Severe Combined Immune Deficiency Patient: a First Report.

Authors:  Monia Ouederni; Fethi Mellouli; Monia Ben Khaled; Houda Kaabi; Capucine Picard; Mohamed Bejaoui
Journal:  J Clin Immunol       Date:  2016-05-05       Impact factor: 8.317

5.  NADPH oxidase correction by mRNA transfection of apheresis granulocytes in chronic granulomatous disease.

Authors:  Suk See De Ravin; Julie Brault; Ronald J Meis; Linhong Li; Narda Theobald; Aylin C Bonifacino; Hong Lei; Taylor Q Liu; Sherry Koontz; Cristina Corsino; Marissa A Zarakas; Jigar V Desai; Aaron B Clark; Uimook Choi; Mark E Metzger; Kamille West; Steven L Highfill; Elizabeth Kang; Douglas B Kuhns; Michail S Lionakis; David F Stroncek; Cynthia E Dunbar; John F Tisdale; Robert E Donahue; Gary A Dahl; Harry L Malech
Journal:  Blood Adv       Date:  2020-12-08

Review 6.  Infectious Complications in Patients With Chronic Granulomatous Disease.

Authors:  Nicholas Bennett; Paul J Maglione; Benjamin L Wright; Christa Zerbe
Journal:  J Pediatric Infect Dis Soc       Date:  2018-05-09       Impact factor: 3.164

7.  Transplantation of Hematopoietic Stem Cells for Primary Immunodeficiencies in Brazil: Challenges in Treating Rare Diseases in Developing Countries.

Authors:  Juliana Folloni Fernandes; Samantha Nichele; Liane E Daudt; Rita B Tavares; Adriana Seber; Fábio R Kerbauy; Adriana Koliski; Gisele Loth; Ana K Vieira; Luiz G Darrigo-Junior; Vanderson Rocha; Alessandra A Gomes; Vergílio Colturato; Luiz F Mantovani; Andreza F Ribeiro; Lisandro L Ribeiro; Cilmara Kuwahara; Ana L M Rodrigues; Victor G Zecchin; Beatriz T Costa-Carvalho; Magda Carneiro-Sampaio; Antonio Condino-Neto; Anders Fasth; Andrew Gennery; Ricardo Pasquini; Nelson Hamerschlak; Carmem Bonfim
Journal:  J Clin Immunol       Date:  2018-11-24       Impact factor: 8.317

8.  Failure to Prevent Severe Graft-Versus-Host Disease in Haploidentical Hematopoietic Cell Transplantation with Post-Transplant Cyclophosphamide in Chronic Granulomatous Disease.

Authors:  Mark Parta; Dianne Hilligoss; Corin Kelly; Nana Kwatemaa; Narda Theobald; Christa S Zerbe; Steven M Holland; Harry L Malech; Elizabeth M Kang
Journal:  J Clin Immunol       Date:  2020-04-20       Impact factor: 8.317

Review 9.  T Cell-Depleted and T Cell-Replete HLA-Haploidentical Stem Cell Transplantation for Non-malignant Disorders.

Authors:  Alice Bertaina; Angela Pitisci; Matilde Sinibaldi; Mattia Algeri
Journal:  Curr Hematol Malig Rep       Date:  2017-02       Impact factor: 3.952

10.  Haploidentical Related Donor Hematopoietic Stem Cell Transplantation for Dedicator-of-Cytokinesis 8 Deficiency Using Post-Transplantation Cyclophosphamide.

Authors:  Nirali N Shah; Alexandra F Freeman; Helen Su; Kristen Cole; Mark Parta; Niki M Moutsopoulos; Safa Baris; Elif Karakoc-Aydiner; Thomas E Hughes; Heidi H Kong; Steve M Holland; Dennis D Hickstein
Journal:  Biol Blood Marrow Transplant       Date:  2017-03-10       Impact factor: 5.742

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