Haematopoietic Stem Cell Transplant for Norovirus-Induced Intestinal Failure in X-linked Agammaglobulinemia.


Journal

Journal of clinical immunology
ISSN: 1573-2592
Titre abrégé: J Clin Immunol
Pays: Netherlands
ID NLM: 8102137

Informations de publication

Date de publication:
10 2021
Historique:
received: 30 04 2021
accepted: 14 06 2021
pubmed: 25 6 2021
medline: 22 2 2022
entrez: 24 6 2021
Statut: ppublish

Résumé

Since the first clinical description in 1952, immunoglobulin replacement therapy remains the mainstay of treatment of patients with X-linked agammaglobulinemia (XLA). However, this therapy only replaces IgG isotype and does not compensate for the loss of Bruton tyrosine kinase in non-B-lymphocytes. Patients may still therefore develop complications despite current standard of care. Here, we describe an XLA patient with persistent chronic norovirus infection, refractory to treatment and causing intestinal failure. The patient underwent haematopoietic stem cell transplantation, curing XLA and allowed clearance of norovirus prior to humoral immunoreconstitution, suggesting non-humoral immunodeficiency in these patients.

Identifiants

pubmed: 34164761
doi: 10.1007/s10875-021-01088-2
pii: 10.1007/s10875-021-01088-2
pmc: PMC8221090
doi:

Types de publication

Case Reports Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

1574-1581

Informations de copyright

© 2021. The Author(s).

Références

Bruton OC. Agammaglobulinemia. Pediatrics. 1952;9(6):722–8.
pubmed: 14929630
Carne E, Ponsford M, El-Shanawany T, Williams P, Pickersgill T, Jolles S. Five years of self-administered hyaluronidase facilitated subcutaneous immunoglobulin (fSCIg) home therapy in a patient with primary immunodeficiency: Figure 1. J Clin Pathol. 2015;69(1):jclinpath-2015–202901–3.
Jolles S, Orange JS, Gardulf A, Stein MR, Shapiro R, Borte M, et al. Current treatment options with immunoglobulin G for the individualization of care in patients with primary immunodeficiency disease. Clin Exp Immunol. 2015;179(2):146–60.
doi: 10.1111/cei.12485
Conley ME, Rohrer J, Rapalus L, Boylin EC, Minegishi Y. Defects in early B-cell development: comparing the consequences of abnormalities in pre-BCR signaling in the human and the mouse. Immunol Rev. 2000;178(1):75–90.
doi: 10.1034/j.1600-065X.2000.17809.x
LoGalbo PR, Sampson HA, Buckley RH. Symptomatic giardiasis in three patients with X-linked agammaglobulinemia. J Pediatr. 1982;101(1):78–80.
doi: 10.1016/S0022-3476(82)80188-1
Linnemann CC Jr, May DB, Schubert WK, Caraway CT, Schiff GM. Fatal viral encephalitis in children with X-linked hypogammaglobulinemia. Am J Dis Child. 1973;126(1):100–3.
pubmed: 4124835
Farrar JE, Rohrer J, Conley ME. Neutropenia in X-linked agammaglobulinemia. Clin Immunol Immunopathol. 1996;81(3):271–6.
doi: 10.1006/clin.1996.0188
Conley ME, Rohrer J, Minegishi Y. X-linked agammaglobulinemia. Clin Rev Allergy Immunol. 2000;19(2):183–204.
doi: 10.1385/CRIAI:19:2:183
Lougaris V, Soresina A, Baronio M, Montin D, Martino S, Signa S, et al. Long-term follow-up of 168 patients with X-linked agammaglobulinemia reveals increased morbidity and mortality. J Allergy Clin Immunol. 2020;146(2):429–37.
doi: 10.1016/j.jaci.2020.03.001
Sperlich JM, Grimbacher B, Workman S, Haque T, Seneviratne SL, Burns SO, et al. Respiratory infections and antibiotic usage in common variable immunodeficiency. J Allergy Clin Immunol Pract. 2018;6(1):159-168.e3.
doi: 10.1016/j.jaip.2017.05.024
Woodward J, Gkrania-Klotsas E, Kumararatne D. Chronic norovirus infection and common variable immunodeficiency. Vol. 188, Clinical and Experimental Immunology. Blackwell Publishing Ltd; 2017. p. 363–70.
Kempf B, Edgar JD, Mc Caughey C, Devlin LA. Nitazoxanide is an ineffective treatment of chronic norovirus in patients with X-linked agammaglobulinemia and may yield false-negative polymerase chain reaction findings in stool specimens. J Infect Dis. 2017;215(3):486–7.
doi: 10.1093/infdis/jiw497
Aadam Z, Kechout N, Barakat A, Chan K-W, Ben-Ali M, Ben-Mustapha L, et al. X-linked agammagobulinemia in a large series of North African patients: frequency, clinical features and novel BTK mutations. J Clin Immunol. 2016.
Brown L-AKAK, Clark I, Brown JR, Breuer J, David L, Lowe M, et al. Norovirus infection in primary immune deficiency. Rev Med Virol. 2017;27(3):e1926.
doi: 10.1002/rmv.1926
De Ravin SS, Wu X, Moir S, Anaya-Obrien S, Kwatemaa N, Littel P, et al. Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency. Sci Transl Med. 2016;8:335ra57.
doi: 10.1126/scitranslmed.aad8856
Huang X, Shi H, Guo N, Lu D. [Allogeneic bone marrow transplantation for the treatment of Bruton disease] (Chinese). Zhonghua Xue Ye Xue Za Zhi. 2002;23:528–30.
pubmed: 12482350
Wan D, Liu C, Wang G, Sun H, Sun L, Jiang Z, et al. [Successful treatment of agammaglobulinemia by HLA-mismatched unrelated cord blood stem cell transplantation–the first case report] (Chinese). Zhonghua Xue Ye Xue Za Zhi. 2005;26:401–3.
pubmed: 16251019
Abu-Arja RF, Chernin LR, Abusin G, Auletta J, Cabral L, Egler R, et al. Successful hematopoietic cell transplantation in a patient with X-linked agammaglobulinemia and acute myeloid leukemia. Pediatr Blood Cancer. 2015;62(9):1674–6.
doi: 10.1002/pbc.25554
Ikegame K, Imai K, Yamashita M, Hoshino A, Kanegane H, Morio T, et al. Allogeneic stem cell transplantation for X-linked agammaglobulinemia using reduced intensity conditioning as a model of the reconstitution of humoral immunity. J Hematol Oncol. 2016;9:9.
doi: 10.1186/s13045-016-0240-y
van Zelm MC, Pumar M, Shuttleworth P, Aui PM, Smart JM, Grigg A, et al. Functional antibody responses following allogeneic stem cell transplantation for TP53 mutant pre-B-ALL in a patient with X-linked agammaglobulinemia. Front Immunol. 2019;10
Nie L, Su T, Yang K-T, Zhao L, Hu J, Yang S-H, et al. Peripheral blood stem cell transplantation from HLA-mismatched unrelated donor or haploidentical donor for the treatment of X-linked agammaglobulinemia. Zhongguo Dang Dai Er Ke Za Zhi Chin J Contemp Pediatr. 2020;22(8):821–7.
Swaminathan VV, Uppuluri R, Patel S, Ramanan KM, Ravichandran N, Jayakumar L, et al. Treosulfan-based reduced toxicity hematopoietic stem cell transplantation in X-linked agammaglobulinemia: a cost-effective alternative to long-term immunoglobulin replacement in developing countries. Pediatr Transplant. 2020;24(1):e13625.
Rawat A, Jindal AK, Suri D, Vignesh P, Gupta A, Saikia B, et al. Clinical and genetic profile of X-linked agammaglobulinemia: a multicenter experience from India. Front Immunol. 2021;11.
Bucciol G, Tousseyn T, Jansen K, Casteels I, Tangye SG, Breuer J, et al. Hematopoietic stem cell transplantation cures chronic Aichi virus infection in a patient with X-linked agammaglobulinemia. J Clin Immunol. 2021 (in press)
Pierce SK, Morris JF, Grusby MJ, Kaumaya P, VAN Buskirk A, Srinivasan M, et al. Antigen-presenting function of B lymphocytes. Immunol Rev. 1988;106(1):149–80.
doi: 10.1111/j.1600-065X.1988.tb00778.x
Bao Y, Zheng J, Han C, Jin J, Han H, Liu Y, et al. Tyrosine kinase Btk is required for NK cell activation. J Biol Chem. 2012;287(28):23769–78.
doi: 10.1074/jbc.M112.372425
Brunner C, Müller B, Wirth T. Bruton’s tyrosine kinase is involved in innate and adaptive immunity. Histol Histopathol. 2005;20:945–55.
pubmed: 15944945
Luk ADW, Ni K, Wu Y, Lam KT, Chan KW, Lee PP, et al. Type I and III interferon productions are impaired in X-linked agammaglobulinemia patients toward poliovirus but not influenza virus. Front Immunol. 2018;9(AUG).
Bucciol G, Moens L, Payne K, Wollants E, Mekahli D, Levtchenko E, et al. Chronic Aichi virus infection in a patient with X-linked agammaglobulinemia. J Clin Immunol. 2018;38(7):748–52.
doi: 10.1007/s10875-018-0558-z
Ponsford MJ, Price C, Farewell D, Greene G, Moore C, Perry M, et al. Increased respiratory viral detection and symptom burden among patients with primary antibody deficiency: results from the BIPAD study. J Allergy Clin Immunol Pract. 2020.
Barmettler S, Otani IM, Minhas J, Abraham RS, Chang Y, Dorsey MJ, et al. Gastrointestinal manifestations in X-linked agammaglobulinemia. J Clin Immunol. 2017;37(3):287–94.
doi: 10.1007/s10875-017-0374-x
Prevot J, Jolles S. Global immunoglobulin supply: steaming towards the iceberg? Curr Opin Allergy Clin Immunol. 2020;20(6):557–64.
doi: 10.1097/ACI.0000000000000696
Chiesa R, Wang J, Blok HJ, Hazelaar S, Neven B, Moshous D, et al. Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adults. Blood. 2020;136(10):1201–11.
doi: 10.1182/blood.2020005590
Cole T, McKendrick F, Titman P, Cant AJ, Pearce MS, Cale CM, et al. Health related quality of life and emotional health in children with chronic granulomatous disease: a comparison of those managed conservatively with those that have undergone haematopoietic stem cell transplant. J Clin Immunol. 2013;33(1):8–13.
doi: 10.1007/s10875-012-9758-0
Bryan BA, Battersby A, Shillitoe BM, Barge D, Bourne H, Flood T, et al. Respiratory health and related quality of life in patients with congenital agammaglobulinemia in the northern region of the UK. J Clin Immunol. 2016;36(5):472–9.
doi: 10.1007/s10875-016-0284-3

Auteurs

Ben M J Shillitoe (BMJ)

Paediatric Immunology, Newcastle Upon Tyne Hospital Trusts, Great North Children's Hospital, Clinical Resource Building, Floor 4, Block 2, Queen Victoria Road, Newcastle upon Tyne, NE1 4LP, UK.

Mark Ponsford (M)

Immunodeficiency Centre for Wales, Cardiff and Vale University Health Board, University Hospital Wales, Cardiff, UK.

Mary A Slatter (MA)

Paediatric Immunology, Newcastle Upon Tyne Hospital Trusts, Great North Children's Hospital, Clinical Resource Building, Floor 4, Block 2, Queen Victoria Road, Newcastle upon Tyne, NE1 4LP, UK.
Translational and Clinical Research Institute, Newcastle Upon Tyne University, Newcastle upon Tyne, UK.

Jennifer Evans (J)

Immunodeficiency Centre for Wales, Cardiff and Vale University Health Board, University Hospital Wales, Cardiff, UK.

Siske Struik (S)

Immunodeficiency Centre for Wales, Cardiff and Vale University Health Board, University Hospital Wales, Cardiff, UK.

Mike Cosgrove (M)

Department of Paediatric Gastroenterology, Singleton Hospital, Swansea, Wales, UK.

Iolo Doull (I)

Department of Paediatric Respiratory Medicine, Cardiff and Vale University Health Board, University Hospital Wales, Cardiff, UK.

Stephen Jolles (S)

Immunodeficiency Centre for Wales, Cardiff and Vale University Health Board, University Hospital Wales, Cardiff, UK.

Andrew R Gennery (AR)

Paediatric Immunology, Newcastle Upon Tyne Hospital Trusts, Great North Children's Hospital, Clinical Resource Building, Floor 4, Block 2, Queen Victoria Road, Newcastle upon Tyne, NE1 4LP, UK. andrew.gennery@ncl.ac.uk.
Translational and Clinical Research Institute, Newcastle Upon Tyne University, Newcastle upon Tyne, UK. andrew.gennery@ncl.ac.uk.

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