Exagamglogene Autotemcel for Severe Sickle Cell Disease

H Frangoul, F Locatelli, A Sharma… - … England Journal of …, 2024 - Mass Medical Soc
Background Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to
reactivate fetal hemoglobin synthesis by means of ex vivo clustered regularly interspaced …

[HTML][HTML] Treatment of sickle cell disease-options and perspective

L Abdel-Hadi, YV Carmenate… - American Journal of …, 2023 - ncbi.nlm.nih.gov
Abstract Sickle Cell Disease (SCD) is one of the most inherited hematologic diseases
affecting humans. Clinically, there is a progressive multiorgan failure and increased mortality …

Pharmacotherapeutical strategies in the prevention of acute, vaso-occlusive pain in sickle cell disease: a systematic review

JWR Sins, DJ Mager, SCAT Davis, BJ Biemond… - Blood …, 2017 - ashpublications.org
Sickle-cell disease (SCD) is characterized by frequent and painful vaso-occlusive crises
(VOCs). Various treatments have been evaluated over the years. However, a clear overview …

Beyond hydroxyurea: new and old drugs in the pipeline for sickle cell disease

MJ Telen - Blood, The Journal of the American Society of …, 2016 - ashpublications.org
Abstract Despite Food and Drug Administration (FDA) approval of hydroxyurea to reduce the
frequency of vaso-occlusive episodes, sickle cell disease (SCD) has continued to be treated …

Vaso-occlusive crisis in sickle cell disease: a vicious cycle of secondary events

T Jang, M Poplawska, E Cimpeanu, G Mo… - Journal of Translational …, 2021 - Springer
Painful vaso-occlusive crisis (VOC) remains the most common reason for presenting to the
Emergency Department and hospitalization in patients with sickle cell disease (SCD) …

The vaso‐occlusive pain crisis in sickle cell disease: definition, pathophysiology, and management

DS Darbari, VA Sheehan… - European journal of …, 2020 - Wiley Online Library
Early diagnosis, treatment, and prevention of a vaso‐occlusive crisis (VOC) are critical to the
management of patients with sickle cell disease. It is essential to differentiate between VOC …

Sickle cell disease complications: Prevalence and resource utilization

N Shah, M Bhor, L Xie, J Paulose, H Yuce - PLoS One, 2019 - journals.plos.org
Objectives This study evaluated the prevalence rate of vaso-occlusive crisis (VOC)
episodes, rates of uncomplicated and complicated VOC episodes, and the primary reasons …

[HTML][HTML] The impact of voxelotor treatment on leg ulcers in patients with sickle cell disease

CP Minniti, J Knight‐Madden, M Tonda… - American journal of …, 2021 - ncbi.nlm.nih.gov
2. Rummel MJ, Niederle N, Maschmeyer G, et al. Bendamustine plus rituximab versus
CHOP plus rituximab as first-line treatment for patients with indolent and mantle-cell …

[HTML][HTML] Initial results from a cohort in a phase 2a study (GBT440-007) evaluating adolescents with sickle cell disease treated with multiple doses of GBT440, a HbS …

CC Hoppe, AC Inati, C Brown, W Wang, VR Gordeuk… - Blood, 2017 - Elsevier
Background: Sickle cell disease (SCD) is a genetic disorder in which deoxygenation
produces polymerization of mutated hemoglobin S (HbS) and triggers the downstream …

Management of sickle cell disease: recommendations from the 2014 expert panel report

BP Yawn, J John-Sowah - American family physician, 2015 - aafp.org
Family physicians are the primary and sometimes only health care resource for families
affected by sickle cell disease. Recently published guidelines provide important …