Acute graft-versus-host disease—biologic process, prevention, and therapy

R Zeiser, BR Blazar - New England Journal of Medicine, 2017 - Mass Medical Soc
When T cells from a bone marrow donor begin to attack the host within 3 months after
hematopoietic-cell transplantation, acute graft-versus-host disease results. The disease …

EBMT/ESID inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity

AC Lankester, MH Albert, C Booth… - Bone Marrow …, 2021 - nature.com
Inborn errors of immunity (IEI) are a group of rare heterogeneous diseases. Currently, more
than 400 monogenetic IEI have been identified and increasingly a genetic diagnosis can be …

Indications for haematopoietic stem cell transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2019

RF Duarte, M Labopin, P Bader, GW Basak… - Bone marrow …, 2019 - nature.com
This is the seventh special EBMT report on the indications for haematopoietic stem cell
transplantation for haematological diseases, solid tumours and immune disorders. Our aim …

Lentiviral gene therapy for X-linked chronic granulomatous disease

DB Kohn, C Booth, EM Kang, SY Pai, KL Shaw… - Nature medicine, 2020 - nature.com
Chronic granulomatous disease (CGD) is a rare inherited disorder of phagocytic cells,. We
report the initial results of nine severely affected X-linked CGD (X-CGD) patients who …

A review of chronic granulomatous disease

DE Arnold, JR Heimall - Advances in therapy, 2017 - Springer
Chronic granulomatous disease (CGD) is a primary immunodeficiency caused by defects in
any of the five subunits of the NADPH oxidase complex responsible for the respiratory burst …

Primary immunodeficiency and autoimmunity: a comprehensive review

L Amaya-Uribe, M Rojas, G Azizi, JM Anaya… - Journal of …, 2019 - Elsevier
The primary immunodeficiency diseases (PIDs) include many genetic disorders that affect
different components of the innate and adaptive responses. The number of distinct genetic …

Hematopoietic stem cell transplantation in primary immunodeficiency diseases: current status and future perspectives

R Castagnoli, OM Delmonte, E Calzoni… - Frontiers in …, 2019 - frontiersin.org
Primary immunodeficiencies (PID) are disorders that for the most part result from mutations
in genes involved in immune host defense and immunoregulation. These conditions are …

Chronic granulomatous disease: a comprehensive review

HH Yu, YH Yang, BL Chiang - Clinical reviews in allergy & immunology, 2021 - Springer
Chronic granulomatous disease (CGD) is a primary immunodeficiency of phagocyte function
due to defective NADPH oxidase (phox). Compared with the common types of CYBB/gp91 …

Association of busulfan exposure with survival and toxicity after haemopoietic cell transplantation in children and young adults: a multicentre, retrospective cohort …

IH Bartelink, A Lalmohamed, EML van Reij… - The Lancet …, 2016 - thelancet.com
Background Intravenous busulfan combined with therapeutic drug monitoring to guide
dosing improves outcomes after allogeneic haemopoietic cell transplantation (HCT). The …

Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adults

R Chiesa, J Wang, HJ Blok, S Hazelaar… - Blood, The Journal …, 2020 - ashpublications.org
Chronic granulomatous disease (CGD) is a primary immunodeficiency resulting in life-
threatening infections and inflammatory complications. Allogeneic hematopoietic cell …