Tandem Orthotopic Living Donor Liver Transplantation Followed by Same Donor Haploidentical Hematopoietic Stem Cell Transplantation for DOCK8 Deficiency.
Adult
Calcineurin Inhibitors
/ administration & dosage
Child
Cryptosporidiosis
/ immunology
Cryptosporidium
/ immunology
Female
Guanine Nucleotide Exchange Factors
/ deficiency
Hematopoietic Stem Cell Transplantation
/ methods
Humans
Liver Transplantation
/ methods
Living Donors
Mothers
Myeloablative Agonists
/ administration & dosage
Severe Combined Immunodeficiency
/ genetics
Transplantation Conditioning
/ methods
Transplantation, Haploidentical
/ methods
Treatment Outcome
Journal
Transplantation
ISSN: 1534-6080
Titre abrégé: Transplantation
Pays: United States
ID NLM: 0132144
Informations de publication
Date de publication:
10 2019
10 2019
Historique:
pubmed:
6
2
2019
medline:
20
6
2020
entrez:
6
2
2019
Statut:
ppublish
Résumé
An 11-year-old girl with dedicator of cytokinesis 8 (DOCK8) deficiency was proposed for potentially curative hematopoietic stem cell transplantation (HSCT), the donor being her haploidentical mother. However, end-stage liver disease caused by chronic Cryptosporidium infection required liver transplantation before HSCT. Consequently, a staged approach of a sequential liver transplant followed by a HSCT was planned with her mother as the donor for both liver and HSCT. The patient successfully underwent a left-lobe orthotopic liver transplant; however, she developed a biliary leak delaying the HSCT. Notably, the recipient demonstrated 3% donor lymphocyte chimerism in her peripheral blood immediately before HSCT. Haploidentical-related donor HSCT performed 2 months after liver transplantation was complicated by the development of acyclovir-resistant herpes simplex virus viremia, primary graft failure, and sinusoidal obstruction syndrome. The patient died from sinusoidal obstruction syndrome-associated multiorgan failure with Candida sepsis on day +40 following HSCT. We discuss the many considerations inherent to planning for HSCT preceded by liver transplant in patients with primary immunodeficiencies, including the role of prolonged immunosuppression and the risk of infection before immune reconstitution. We also discuss the implications of potential recipient sensitization against donor stem cells precipitated by exposure of the recipient to the donor lymphocytes from the transplanted organ.
Sections du résumé
BACKGROUND
An 11-year-old girl with dedicator of cytokinesis 8 (DOCK8) deficiency was proposed for potentially curative hematopoietic stem cell transplantation (HSCT), the donor being her haploidentical mother. However, end-stage liver disease caused by chronic Cryptosporidium infection required liver transplantation before HSCT.
METHODS
Consequently, a staged approach of a sequential liver transplant followed by a HSCT was planned with her mother as the donor for both liver and HSCT.
RESULTS
The patient successfully underwent a left-lobe orthotopic liver transplant; however, she developed a biliary leak delaying the HSCT. Notably, the recipient demonstrated 3% donor lymphocyte chimerism in her peripheral blood immediately before HSCT. Haploidentical-related donor HSCT performed 2 months after liver transplantation was complicated by the development of acyclovir-resistant herpes simplex virus viremia, primary graft failure, and sinusoidal obstruction syndrome. The patient died from sinusoidal obstruction syndrome-associated multiorgan failure with Candida sepsis on day +40 following HSCT.
CONCLUSIONS
We discuss the many considerations inherent to planning for HSCT preceded by liver transplant in patients with primary immunodeficiencies, including the role of prolonged immunosuppression and the risk of infection before immune reconstitution. We also discuss the implications of potential recipient sensitization against donor stem cells precipitated by exposure of the recipient to the donor lymphocytes from the transplanted organ.
Identifiants
pubmed: 30720689
doi: 10.1097/TP.0000000000002649
pmc: PMC6667308
mid: NIHMS1519614
doi:
Substances chimiques
Calcineurin Inhibitors
0
DOCK8 protein, human
0
Guanine Nucleotide Exchange Factors
0
Myeloablative Agonists
0
Banques de données
ClinicalTrials.gov
['NCT01176006']
Types de publication
Case Reports
Journal Article
Research Support, N.I.H., Extramural
Research Support, N.I.H., Intramural
Langues
eng
Sous-ensembles de citation
IM
Pagination
2144-2149Subventions
Organisme : CCR NIH HHS
ID : HHSN261200800001C
Pays : United States
Organisme : NCI NIH HHS
ID : HHSN261200800001E
Pays : United States
Organisme : Intramural NIH HHS
ID : Z99 CA999999
Pays : United States
Références
Am J Surg Pathol. 1999 Jun;23(6):734-7
pubmed: 10366158
N Engl J Med. 2000 Feb 3;342(5):320-4
pubmed: 10655530
Blood. 2000 Dec 1;96(12):3997-9
pubmed: 11090093
Bone Marrow Transplant. 1992 Oct;10(4):367-72
pubmed: 1422493
J Pediatr. 2004 Sep;145(3):333-9
pubmed: 15343186
J Allergy Clin Immunol. 2008 Dec;122(6):1087-96
pubmed: 18992926
Blood. 1991 Aug 15;78(4):1140-3
pubmed: 1907874
Biol Blood Marrow Transplant. 2010 Feb;16(2):157-68
pubmed: 19766729
N Engl J Med. 2009 Nov 19;361(21):2046-55
pubmed: 19776401
Clin Transplant. 2009 Dec;23 Suppl 21:37-41
pubmed: 19930315
Cell Ther Transplant. 2010 Aug 31;2(8):null
pubmed: 21152385
Exp Hematol. 2012 Jul;40(7):513-7
pubmed: 22542577
Biol Blood Marrow Transplant. 2012 Nov;18(11):1629-37
pubmed: 22892552
Clin Rev Allergy Immunol. 2014 Apr;46(2):131-44
pubmed: 23832379
Curr Opin Organ Transplant. 2013 Aug;18(4):402-7
pubmed: 23838644
Clin Immunol. 2014 May-Jun;152(1-2):111-4
pubmed: 24667686
Bone Marrow Transplant. 2014 Aug;49(8):999-1008
pubmed: 24842530
Biol Blood Marrow Transplant. 2014 Oct;20(10):1573-9
pubmed: 24910379
N Engl J Med. 2014 Jul 31;371(5):434-46
pubmed: 25075835
J Clin Immunol. 2015 Feb;35(2):189-98
pubmed: 25627830
Biol Blood Marrow Transplant. 2015 Jun;21(6):1037-45
pubmed: 25636378
J Clin Exp Hepatol. 2014 Dec;4(4):332-46
pubmed: 25755580
Biol Blood Marrow Transplant. 2015 Jul;21(7):1299-307
pubmed: 25797174
Blood. 2016 Mar 31;127(13):1656-65
pubmed: 26825712
Biol Blood Marrow Transplant. 2016 May;22(5):895-901
pubmed: 26860634
J Allergy Clin Immunol. 2016 Sep;138(3):852-859.e3
pubmed: 27130861
Bone Marrow Transplant. 2016 Jul;51(7):906-12
pubmed: 27183098
Adv Hematol. 2016;2016:6471901
pubmed: 27239198
J Allergy Clin Immunol Pract. 2016 Nov - Dec;4(6):1239-1242.e1
pubmed: 27641484
Clin Immunol. 2017 May;178:39-44
pubmed: 27890707
Biol Blood Marrow Transplant. 2017 Jun;23(6):997-1004
pubmed: 28285079
Biol Blood Marrow Transplant. 2017 Jun;23(6):980-990
pubmed: 28288951
Pediatr Transplant. 2017 Nov;21(7):null
pubmed: 28664550
J Clin Virol. 2017 Oct;95:20-25
pubmed: 28837858
Pediatr Blood Cancer. 2018 Jan;65(1):null
pubmed: 28901730
Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):645-650
pubmed: 29222316
Bone Marrow Transplant. 2018 Mar;53(3):339-343
pubmed: 29269803
Blood Adv. 2017 Feb 14;1(6):397-400
pubmed: 29296954
Transplantation. 2018 May;102(5):823-828
pubmed: 29377874
Transpl Int. 2018 Jun;31(6):639-648
pubmed: 29464765
Blood Adv. 2018 Mar 13;2(5):575-585
pubmed: 29535106
Hepatology. 2019 Feb;69(2):917-919
pubmed: 30151988
Hepatology. 1993 Jun;17(6):1127-52
pubmed: 8514264