Long-term outcome of hematopoietic stem cell transplantation for IL2RG/JAK3 SCID: a cohort report.
Identifieur interne : 008D44 ( Ncbi/Curation ); précédent : 008D43; suivant : 008D45Long-term outcome of hematopoietic stem cell transplantation for IL2RG/JAK3 SCID: a cohort report.
Auteurs : Intan Juliana Abd Hamid [Royaume-Uni] ; Mary A. Slatter [Royaume-Uni] ; Fiona Mckendrick ; Mark S. Pearce [Royaume-Uni] ; Andrew R. Gennery [Royaume-Uni]Source :
- Blood [ 1528-0020 ] ; 2017.
Descripteurs français
- KwdFr :
- Adolescent, Adulte, Allogreffes, Antigènes CD (génétique), Antigènes CD (immunologie), Chimère obtenue par transplantation (génétique), Chimère obtenue par transplantation (immunologie), Enfant, Enfant d'âge préscolaire, Facteurs temps, Femelle, Humains, Immunodéficience combinée grave (), Immunodéficience combinée grave (génétique), Immunodéficience combinée grave (immunologie), Immunodéficience combinée grave (mortalité), Janus kinase 3, Lymphocytes T (immunologie), Mâle, Qualité de vie, Sous-unité gamma commune aux récepteurs des interleukines (génétique), Survie sans rechute, Taux de survie, Transplantation de cellules souches hématopoïétiques, Études de suivi, Études rétrospectives.
- MESH :
- génétique : Antigènes CD, Chimère obtenue par transplantation, Immunodéficience combinée grave, Sous-unité gamma commune aux récepteurs des interleukines.
- immunologie : Antigènes CD, Chimère obtenue par transplantation, Immunodéficience combinée grave, Lymphocytes T.
- mortalité : Immunodéficience combinée grave.
- Adolescent, Adulte, Allogreffes, Enfant, Enfant d'âge préscolaire, Facteurs temps, Femelle, Humains, Immunodéficience combinée grave, Janus kinase 3, Mâle, Qualité de vie, Survie sans rechute, Taux de survie, Transplantation de cellules souches hématopoïétiques, Études de suivi, Études rétrospectives.
English descriptors
- KwdEn :
- Adolescent, Adult, Allografts, Antigens, CD (genetics), Antigens, CD (immunology), Child, Child, Preschool, Disease-Free Survival, Female, Follow-Up Studies, Hematopoietic Stem Cell Transplantation, Humans, Interleukin Receptor Common gamma Subunit (genetics), Janus Kinase 3, Male, Quality of Life, Retrospective Studies, Severe Combined Immunodeficiency (genetics), Severe Combined Immunodeficiency (immunology), Severe Combined Immunodeficiency (mortality), Severe Combined Immunodeficiency (therapy), Survival Rate, T-Lymphocytes (immunology), Time Factors, Transplantation Chimera (genetics), Transplantation Chimera (immunology).
- MESH :
- chemical , genetics : Antigens, CD, Interleukin Receptor Common gamma Subunit.
- chemical , immunology : Antigens, CD.
- genetics : Severe Combined Immunodeficiency, Transplantation Chimera.
- immunology : Severe Combined Immunodeficiency, T-Lymphocytes, Transplantation Chimera.
- mortality : Severe Combined Immunodeficiency.
- therapy : Severe Combined Immunodeficiency.
- Adolescent, Adult, Allografts, Child, Child, Preschool, Disease-Free Survival, Female, Follow-Up Studies, Hematopoietic Stem Cell Transplantation, Humans, Janus Kinase 3, Male, Quality of Life, Retrospective Studies, Survival Rate, Time Factors.
Abstract
Hematopoietic stem cell transplantation (HSCT) cures the T-lymphocyte, B-lymphocyte, and natural killer (NK)-cell differentiation defect in interleukin-2 γ-chain receptor (IL2RG)/JAK3 severe combined immunodeficiency (SCID). We evaluated long-term clinical features, longitudinal immunoreconstitution, donor chimerism, and quality of life (QoL) of IL2RG/JAK3 SCID patients >2 years post-HSCT at our center. Clinical data were collated and patients/families answered PedsQL Generic Core Scale v4.0 questionnaires. We performed longitudinal analyses of CD3(+), CD4(+) naive T-lymphocyte, CD19(+), and NK-cell numbers from pretransplant until 15 years posttransplant. Thirty-one of 43 patients (72%) survived. Median age at last follow-up was 10 years (range, 2-25 years). Twenty-one (68%) had persistent medical issues, mainly ongoing immunoglobulin replacement (14; 45%), cutaneous viral warts (7; 24%), short stature (4; 14%), limb lymphoedema (3; 10%), and bronchiectasis (2; 7%). Lung function was available and normal for 6 patients. Longitudinal analysis demonstrated sustained CD3(+), CD19(+), and NK-cell output 15 years post-HSCT. CD4(+) naive lymphocyte numbers were better in conditioned vs unconditioned recipients (P, .06). B-lymphocyte and myeloid chimerism were highly correlated (ρ, 0.98; P < .001). Low-toxicity myeloablative conditioning recipients have better B-lymphocyte/myeloid chimerism and are free from immunoglobulin replacement therapy. IL2RG/JAK3 SCID survivors free from immunoglobulin replacement have normal QoL.
DOI: 10.1182/blood-2016-11-748616
PubMed: 28209722
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Fiona Mckendrick<affiliation><nlm:affiliation>Department of Health Psychology, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom; and.</nlm:affiliation>
<wicri:noCountry code="subField">United Kingdom; and</wicri:noCountry>
</affiliation>
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<term>Adult</term>
<term>Allografts</term>
<term>Antigens, CD (genetics)</term>
<term>Antigens, CD (immunology)</term>
<term>Child</term>
<term>Child, Preschool</term>
<term>Disease-Free Survival</term>
<term>Female</term>
<term>Follow-Up Studies</term>
<term>Hematopoietic Stem Cell Transplantation</term>
<term>Humans</term>
<term>Interleukin Receptor Common gamma Subunit (genetics)</term>
<term>Janus Kinase 3</term>
<term>Male</term>
<term>Quality of Life</term>
<term>Retrospective Studies</term>
<term>Severe Combined Immunodeficiency (genetics)</term>
<term>Severe Combined Immunodeficiency (immunology)</term>
<term>Severe Combined Immunodeficiency (mortality)</term>
<term>Severe Combined Immunodeficiency (therapy)</term>
<term>Survival Rate</term>
<term>T-Lymphocytes (immunology)</term>
<term>Time Factors</term>
<term>Transplantation Chimera (genetics)</term>
<term>Transplantation Chimera (immunology)</term>
</keywords>
<keywords scheme="KwdFr" xml:lang="fr"><term>Adolescent</term>
<term>Adulte</term>
<term>Allogreffes</term>
<term>Antigènes CD (génétique)</term>
<term>Antigènes CD (immunologie)</term>
<term>Chimère obtenue par transplantation (génétique)</term>
<term>Chimère obtenue par transplantation (immunologie)</term>
<term>Enfant</term>
<term>Enfant d'âge préscolaire</term>
<term>Facteurs temps</term>
<term>Femelle</term>
<term>Humains</term>
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<term>Immunodéficience combinée grave (génétique)</term>
<term>Immunodéficience combinée grave (immunologie)</term>
<term>Immunodéficience combinée grave (mortalité)</term>
<term>Janus kinase 3</term>
<term>Lymphocytes T (immunologie)</term>
<term>Mâle</term>
<term>Qualité de vie</term>
<term>Sous-unité gamma commune aux récepteurs des interleukines (génétique)</term>
<term>Survie sans rechute</term>
<term>Taux de survie</term>
<term>Transplantation de cellules souches hématopoïétiques</term>
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<term>Études rétrospectives</term>
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<term>Chimère obtenue par transplantation</term>
<term>Immunodéficience combinée grave</term>
<term>Sous-unité gamma commune aux récepteurs des interleukines</term>
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<term>Chimère obtenue par transplantation</term>
<term>Immunodéficience combinée grave</term>
<term>Lymphocytes T</term>
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<keywords scheme="MESH" qualifier="immunology" xml:lang="en"><term>Severe Combined Immunodeficiency</term>
<term>T-Lymphocytes</term>
<term>Transplantation Chimera</term>
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<keywords scheme="MESH" qualifier="mortality" xml:lang="en"><term>Severe Combined Immunodeficiency</term>
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<term>Adult</term>
<term>Allografts</term>
<term>Child</term>
<term>Child, Preschool</term>
<term>Disease-Free Survival</term>
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<term>Follow-Up Studies</term>
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<term>Janus Kinase 3</term>
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<term>Time Factors</term>
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<term>Qualité de vie</term>
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<term>Taux de survie</term>
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<front><div type="abstract" xml:lang="en">Hematopoietic stem cell transplantation (HSCT) cures the T-lymphocyte, B-lymphocyte, and natural killer (NK)-cell differentiation defect in interleukin-2 γ-chain receptor (IL2RG)/JAK3 severe combined immunodeficiency (SCID). We evaluated long-term clinical features, longitudinal immunoreconstitution, donor chimerism, and quality of life (QoL) of IL2RG/JAK3 SCID patients >2 years post-HSCT at our center. Clinical data were collated and patients/families answered PedsQL Generic Core Scale v4.0 questionnaires. We performed longitudinal analyses of CD3(+), CD4(+) naive T-lymphocyte, CD19(+), and NK-cell numbers from pretransplant until 15 years posttransplant. Thirty-one of 43 patients (72%) survived. Median age at last follow-up was 10 years (range, 2-25 years). Twenty-one (68%) had persistent medical issues, mainly ongoing immunoglobulin replacement (14; 45%), cutaneous viral warts (7; 24%), short stature (4; 14%), limb lymphoedema (3; 10%), and bronchiectasis (2; 7%). Lung function was available and normal for 6 patients. Longitudinal analysis demonstrated sustained CD3(+), CD19(+), and NK-cell output 15 years post-HSCT. CD4(+) naive lymphocyte numbers were better in conditioned vs unconditioned recipients (P, .06). B-lymphocyte and myeloid chimerism were highly correlated (ρ, 0.98; P < .001). Low-toxicity myeloablative conditioning recipients have better B-lymphocyte/myeloid chimerism and are free from immunoglobulin replacement therapy. IL2RG/JAK3 SCID survivors free from immunoglobulin replacement have normal QoL.</div>
</front>
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