Modern management of Fanconi anemia

C Dufour, F Pierri - Hematology, 2022 - ashpublications.org
In this review, we present a clinical case report and discussion to outline the importance of
long-term specific Fanconi anemia (FA) monitoring, and we discuss the main aspects of the …

Transplantation for Fanconi anaemia: lessons learned from Brazil

C Bonfim, S Nichele, G Loth, VAM Funke… - The Lancet …, 2022 - thelancet.com
Fanconi anaemia is a challenging disease to manage, and haematopoietic stem-cell
transplantation (HSCT) is the treatment of choice for the haematological complications …

Beyond current treatment of Fanconi Anemia: What do advances in cell and gene-based approaches offer?

E Martínez-Balsalobre, JH Guervilly… - Blood Reviews, 2023 - Elsevier
Fanconi anemia (FA) is a rare inherited disorder that mainly affects the bone marrow. This
condition causes decreased production of all types of blood cells. FA is caused by a …

Recent advances in hematopoietic cell transplantation for inherited bone marrow failure syndromes

H Sakaguchi, N Yoshida - International Journal of Hematology, 2022 - Springer
Inherited bone marrow failure syndromes (IBMFSs) are a group of rare genetic disorders
characterized by bone marrow failure with unique phenotypes and predisposition to cancer …

Hematopoietic stem cell transplantation for classical inherited bone marrow failure syndromes: an update

F Pierri, M Faraci, S Giardino… - Expert Review of …, 2021 - Taylor & Francis
Introduction Inherited bone marrow failure syndromes (IBMFS) feature complex molecular
pathophysiology resulting in ineffective hematopoiesis and increased risk of progression to …

Unmanipulated haploidentical haematopoietic cell transplantation with radiation‐free conditioning in Fanconi anaemia: A retrospective analysis from the Chinese …

L Xu, Y Lu, S Hu, C Li, Y Tang, H Wang… - British Journal of …, 2022 - Wiley Online Library
Haematopoietic cell transplantation (HCT) is the only curative treatment for haematological
complications in patients with Fanconi anaemia (FA). Haploidentical (haplo‐) HCT is a …

Management of Fanconi anemia beyond childhood

TS Olson - Hematology, 2023 - ashpublications.org
Fanconi anemia (FA) has long been considered a severe inherited bone marrow failure
(BMF) disorder of early childhood. Thus, management of this multisystem disorder has …

Current and future perspectives of Fanconi Anemia treatment

E Martínez-Balsalobre, JH Guervilly, AB Pérez-Oliva… - Blood Reviews, 2023 - hal.science
Fanconi anemia (FA) is a rare inherited disorder that mainly affects the bone marrow. This
condition causes decreased production of all types of blood cells. FA is caused by a …

[HTML][HTML] Pediatric Bone Marrow Failure: A Broad Landscape in Need of Personalized Management

LTW Vissers, M van der Burg, AC Lankester… - Journal of Clinical …, 2023 - mdpi.com
Irreversible severe bone marrow failure (BMF) is a life-threatening condition in pediatric
patients. Most important causes are inherited bone marrow failure syndromes (IBMFSs) and …

[HTML][HTML] Umbilical Cord Blood Transplantation for Fanconi Anemia with a special focus on late complications: a Study on Behalf of Eurocord and SAAWP-EBMT

H Rafii, F Volt, M Bierings, JH Dalle, M Ayas… - … and Cellular Therapy, 2024 - Elsevier
Hematopoietic cell transplantation (HCT) remains the sole available curative treatment for
Fanconi anemia (FA), with particularly favorable outcomes reported after matched sibling …