Hematopoietic Cell Transplantation for Immunodeficiency Diseases

Small, Trudy N.; Friedrich, Wilhelm; O’Reilly, Richard J.

doi:10.1002/9780470987070.ch105

Cited by 9 publications

(11 citation statements)

References 151 publications

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“…The current standard of care for ADA-deficient SCID with a human leukocyte antigen (HLA)-matched sibling donor is bone marrow transplantation (BMT), without prior marrow cytoreductive conditioning. 2,3 Allogeneic transplantation with unfractionated, whole marrow from an HLAmatched sibling into an unconditioned SCID recipient usually results in complete and enduring restoration of immunity. The exact mechanisms by which nonmyeloablative allogeneic transplantation leads to immune reconstitution in SCID are not fully understood.…”

Section: Introductionmentioning

confidence: 99%

Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective donor T lymphoid expansion

Carbonaro

Jin

Cotoi

et al. 2008

Blood

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Section: Introductionmentioning

confidence: 99%

Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective donor T lymphoid expansion

Carbonaro

Jin

Cotoi

et al. 2008

Blood

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“…In all patients, these values were below the normal range for age. 16 [1][2][3][4][6][7][8][9][10][11][12] For certain inherited T-cell immunodeficiencies, unconditioned transplantation using HLA-identical grafts can be successful because host cellular immunity is insufficient to cause graft rejection (reviewed in Small et al 17 ). However, in MHC class II deficiency, residual host immunity is sufficient to reject donor grafts in the absence of preconditioning.…”

Section: Chimerismmentioning

confidence: 99%

Alternative donor SCT for the treatment of MHC Class II deficiency

Small

Qasim

Friedrich

et al. 2012

Bone Marrow Transplant

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MHC Class II deficiency is a rare primary immunodeficiency disease characterized by absent HLA Class II expression resulting in CD4 lymphopenia, lack of Ag-specific responses and recurrent infection. Without successful allogeneic SCT, most children succumb to infection within the first decade of life. To date, alternative donor transplants for this disorder have been inferior to SCT for other forms of combined immunodeficiency disease due to an increased incidence of graft rejection, GVHD and death from infections generally acquired before haematopoietic cell transplantation. This study details the transplant outcome of 16 affected children consecutively transplanted at four centers since 1990, 8 of whom required mechanical ventilation pretransplant. Stem cells were derived from an HLA-mismatched family member (n ¼ 10), an HLA-matched unrelated adult donor (n ¼ 4), or an unrelated cord blood donor (n ¼ 2). Graft failure occurred in five children, all of whom underwent a second SCT. Six patients developed acute GVHD although no patient developed chronic GVHD after primary transplantation. CD4 T-cell reconstitution remained below the normal range for age, suggesting defective thymopoiesis after allo-SCT. Nonetheless, 69% of children survive without GVHD at a median follow-up of 5.7 years, indicating improved outcomes compared with previous studies.

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“…Birinci grupta hücresel bağışıklıkta rol oynayan T lenfositler, humoral bağışıklıkta rol oynayan B lenfositler, doğal öldürücü NK hücreleri yer alır. İkinci grupta ise monosit, makrofaj ve bölünmüş çekirdekli beyaz küreler yer alır 1 . Tüm bu primer immün yetmezlik hastalıkları genetik hasarlanmalar sonucunda ortaya çıkmaktadır.…”

Section: Introductionunclassified

“…Tüm bu primer immün yetmezlik hastalıkları genetik hasarlanmalar sonucunda ortaya çıkmaktadır. Sağlıklı uygun allojenik vericilerden HKHN ile hasarlı durumun düzeltilmesi çoğu hastada tedavi sağlamaktadır 1 . Enzim replasman tedavisi ve otolog hematopoetik kök hücreler içerisine gen transferi spesifik immün yetmezliklerde yapılan HKHN' ne alternatif diğer tedavi yaklaşımlarıdır.…”

Section: Introductionunclassified

“…Ağır kombine immün yetmezlik olmayanlar (non-SCID) ise; T hücre yetmezlikleri, CD-40 ligand yetmezliği, Wiskott-Aldrich Sendromu (WAS), X'e bağlı lenfoproliferatif hastalıklar, fagositik hücre hastalıkları, hemofagositik sendromlar ve otoimmün ve immünregulatuar hastalıklar olarak alt gruplara ayrılır 1,2 . Bu yazıda primer immmün yetmezlik hastalıkları için tek küratif tedavi yöntemi olan HKHN ile ilgili bilgiler derlenmiştir.…”

Section: Introductionunclassified

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Primer İmmün Yetmezlik Hastalıkları ve Hematopoetik Kök Hücre Nakli

Özkan¹

2014

Arşiv Kaynak Tarama Dergisi

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Hematopoietic stem cell transplantation is the only curative therapy for primary immunodeficiency diseases. Early diagnosis, including prenatally, and early transplantation improve stem cell transplantation outcomes. Survival rates improve with advances in the methods of preparing hosts and donor cells, and in supportive and conditioning regimes. Key words: Hematopoietic stem cell transplantation, primary immunodeficiency ÖZET Hematopoetik kök hücre nakli birçok primer immün yetmezlik hastalıkları için tek küratif tedavi yöntemidir. Prenatal dönemi kapsayan erken tanı ve erken kök hücre nakli ile daha iyi sonuçlar alınmaktadır. Hazırlık ve destek tedavilerinin, alıcıların ve verici hücrelerinin hazırlanmasındaki metodların geliştirilmesi ile sağkalım oranlarında artış olmaktadır. Anahtar kelimeler: Hematopoetik kök hücre nakli, primer immün yetmezlik GirişBağışıklık sistemi hücrelerinin kökeni, kemik iliğinin kök hücre denilen farklı yönde değişme yeteneği olan hücreleridir. Kök hücrelerden farklılaşarak oluşan hücrelerden immün sistemle ilişkili olanlar iki gruba ayrılır. Birinci grupta hücresel bağışıklıkta rol oynayan T lenfositler, humoral bağışıklıkta rol oynayan B lenfositler, doğal öldürücü NK hücreleri yer alır. İkinci

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Hematopoietic Cell Transplantation for Immunodeficiency Diseases

Cited by 9 publications

References 151 publications

Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective donor T lymphoid expansion

Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective donor T lymphoid expansion

Alternative donor SCT for the treatment of MHC Class II deficiency

Primer İmmün Yetmezlik Hastalıkları ve Hematopoetik Kök Hücre Nakli

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