Sickle cell disease

JB Kunz, L Tagliaferri - Transfusion Medicine and Hemotherapy, 2024 - karger.com
Background: Sickle cell disease (SCD) is among the most frequent hereditary disorders
globally and its prevalence in Europe is increasing due to migration movements. Summary …

Budget Impact of Disease-Modifying Treatments and a CRISPR Gene-Edited Therapy for Sickle Cell Disease

K Abdallah, I Huys, KJ Claes, S Simoens - Clinical Drug Investigation, 2024 - Springer
Abstract Background and Objective Treatment of sickle cell disease (SCD) has traditionally
focused on symptomatic and preventative care. Recent advances in novel therapeutic …

[HTML][HTML] Quality of life and out-of-pocket expenditures for sickle cell disease patients in Saudi Arabia: A Single-Center Study

Y AlRuthia, RB Alanazi, SF Alotaibi, M Alanazi - Healthcare, 2024 - mdpi.com
Background: Sickle cell anemia (SCD) is a relatively uncommon health condition in many
countries, but it is prevalent in Saudi Arabia mainly due to the high incidence of …

[HTML][HTML] Considerations for applying emerging technologies in paediatric laboratory medicine

T Lang, S Geaghan, TP Loh, C Mak… - Clinical Chemistry and …, 2024 - degruyter.com
Emerging technology in laboratory medicine can be defined as an analytical method
(including biomarkers) or device (software, applications, and algorithms) that by its stage of …

The acute pain crisis in sickle cell disease: What can be done to improve outcomes?

P Telfer, KA Anie, S Kotsiopoulou, L Aiken, S Hibbs… - Blood Reviews, 2024 - Elsevier
The acute pain crisis (APC) is the commonest complication of sickle cell disease (SCD).
Severe episodes may require treatment in hospital with strong opioid analgesic drugs …

Haemoglobinopathies and other rare anemias in Spain: ten years of a nationwide registry (REHem-AR)

JMM Sánchez, EJB Cancho, D Benéitez… - Annals of …, 2024 - pmc.ncbi.nlm.nih.gov
REHem-AR was created in 2013. The progressive implementation of neonatal screening for
haemoglobinopathies in Spanish autonomous communities where the registry had not been …

Pharmacogenomics of Drugs Used in β-Thalassemia and Sickle-Cell Disease: From Basic Research to Clinical Applications

R Gambari, AD Waziri, H Goonasekera… - International journal of …, 2024 - mdpi.com
In this short review we have presented and discussed studies on pharmacogenomics (also
termed pharmacogenetics) of the drugs employed in the treatment of β-thalassemia or Sickle …

Trends and outcomes of hospitalized patients with priapism in Germany: results from the GRAND study

N Pyrgidis, GB Schulz, M Chaloupka, Y Volz… - International Journal of …, 2024 - nature.com
We aimed to provide evidence on the trends and in-hospital outcomes of patients with low-
and high-flow priapism through the largest study in the field. We used the GeRmAn …

[HTML][HTML] Newborn Screening for Sickle Cell Disease in Catalonia between 2015 and 2022—Epidemiology and Impact on Clinical Events

JM González de Aledo-Castillo… - International Journal of …, 2024 - mdpi.com
In 2015, Catalonia introduced sickle cell disease (SCD) screening in its newborn screening
(NBS) program along with standard-of-care treatments like penicillin, hydroxyurea, and anti …

Limited access to transcranial Doppler screening and stroke prevention for children with sickle cell disease in Europe: Results of a multinational EuroBloodNet survey

V Voi, V Gutierrez‐Valle, D Cuzzubbo… - Pediatric Blood & …, 2024 - Wiley Online Library
Background Ensuring equitable access to adequate standard of care for patients with rare
hematological disease is one of the aims of the European Reference Network (ERN) …