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 …

Pyrrolizidine alkaloids: potential role in the etiology of cancers, pulmonary hypertension, congenital anomalies, and liver disease

JA Edgar, RJ Molyneux… - Chemical research in …, 2015 - ACS Publications
Large outbreaks of acute food-related poisoning, characterized by hepatic sinusoidal
obstruction syndrome, hemorrhagic necrosis, and rapid liver failure, occur on a regular basis …

BCSH/BSBMT guideline: diagnosis and management of veno‐occlusive disease (sinusoidal obstruction syndrome) following haematopoietic stem cell transplantation

FL Dignan, RF Wynn, N Hadzic, J Karani… - British journal of …, 2013 - Wiley Online Library
Summary of Key Recommendations Diagnosis It is recommended that the diagnosis of veno‐
occlusive disease (sinusoidal obstruction syndrome)[VOD (SOS)] be based primarily on …

[HTML][HTML] Single-center experience of unrelated and haploidentical stem cell transplantation with TCRαβ and CD19 depletion in children with primary immunodeficiency …

D Balashov, A Shcherbina, M Maschan… - Biology of Blood and …, 2015 - Elsevier
The transplantation of stem cells from a matched unrelated donor (MUD) or a haploidentical
mismatched related donor (MMRD) is a widely used variant of curative treatment for patients …

Treosulfan or busulfan plus fludarabine as conditioning treatment before allogeneic haemopoietic stem cell transplantation for older patients with acute myeloid …

DW Beelen, R Trenschel, M Stelljes, C Groth… - The Lancet …, 2020 - thelancet.com
Background Further improvement of preparative regimens before allogeneic haemopoietic
stem cell transplantation (HSCT) is an unmet medical need for the growing number of older …

Wiskott–Aldrich syndrome: diagnosis, current management, and emerging treatments

D Buchbinder, DJ Nugent… - The application of clinical …, 2014 - Taylor & Francis
Wiskott–Aldrich syndrome (WAS) is a rare X-linked primary immunodeficiency disorder
characterized by the triad of eczema, thrombocytopenia, and severe and often recurrent …

Allogeneic hematopoietic stem cell transplantation in thalassemia major: results of a reduced-toxicity conditioning regimen based on the use of treosulfan

ME Bernardo, E Piras, A Vacca… - Blood, The Journal …, 2012 - ashpublications.org
Sixty thalassemia patients (median age, 7 years; range, 1-37) underwent allogeneic
hematopoietic stem cell transplantation (HSCT) after a preparation combining thiotepa …

Treosulfan-based conditioning for allogeneic HSCT in children with chronic granulomatous disease: a multicenter experience

B Morillo-Gutierrez, R Beier, K Rao… - Blood, The Journal …, 2016 - ashpublications.org
Chronic granulomatous disease (CGD) can be cured by allogeneic hemopoietic stem cell
transplantation (HSCT). Complications include graft failure, graft-versus-host disease …

Reduced-intensity conditioning for hematopoietic cell transplant for HLH and primary immune deficiencies

CE Allen, R Marsh, P Dawson… - Blood, The Journal …, 2018 - ashpublications.org
Allogeneic hematopoietic cell transplantation (HCT) with myeloablative conditioning for
disorders associated with excessive inflammation such as hemophagocytic …

Outcome of hematopoietic stem cell transplantation for adenosine deaminase–deficient severe combined immunodeficiency

A Hassan, C Booth, A Brightwell… - Blood, The Journal …, 2012 - ashpublications.org
Deficiency of the purine salvage enzyme adenosine deaminase leads to SCID (ADA-SCID).
Hematopoietic cell transplantation (HCT) can lead to a permanent cure of SCID; however …