Clinical and economic impact of rFVIIa prophylaxis in Glanzmann thrombasthenia: cohort analysis in Saudi Arabia.
6 affiliations
- 1Madinah Hereditary Blood Disorders Center, King Salman Medical City, Madinah, Saudi Arabia.
- 2Maternity and Children Hospital, Al-Ahsa, Saudi Arabia.
- 3King Salman Medical City, Madinah, Saudi Arabia.
- 4Faculty of Medicine, Aswan University, Aswan, Egypt.
- 5DHT.health, Birchwood, United Kingdom.
- 6Health Innovation Campus, Lancaster University, Lancaster, United Kingdom.
Abstract
Glanzmann thrombasthenia (GT) is an ultrarare inherited platelet disorder associated with lifelong bleeding tendency and substantial health care burden. Evidence on the clinical and economic impact of prophylaxis in GT remains limited. We conducted a prospective cohort analysis of pediatric patients with severe GT receiving prophylactic recombinant activated factor VII (rFVIIa) at a specialist tertiary center in Saudi Arabia. Annualized clinical outcomes and direct medical costs during prophylaxis were compared with a historical preprophylaxis period of equal duration for each patient. Outcomes included treated bleeding episodes, hospital days, platelet transfusion use, and rFVIIa consumption. Costs were estimated by applying unit costs to captured resource use. Paired nonparametric analyses and deterministic sensitivity analyses were conducted. Eleven patients were included. Median annualized treated bleeding episodes decreased from 10 during the preprophylaxis period to 4 during prophylaxis. Median annualized hospital days decreased from 96 to 24, whereas platelet transfusion use decreased from 63 to 6 units. Although rFVIIa acquisition costs increased during prophylaxis, these were offset by lower hospitalization and platelet transfusion costs, resulting in a median net annual cost impact of -1305 Saudi riyal (SAR). Deterministic sensitivity analyses demonstrated robustness to plausible variation in costing assumptions, with median net annual cost impact ranging from -93 339 SAR to 90 729 SAR. These findings provide the first evidence indicating clinical and economic benefits associated with rFVIIa prophylaxis in GT and support further evaluation of prophylactic treatment strategies in this population.
Related
- PO084 | BRIDGING THE CLINICAL CHASM: GENETIC ANALYSIS AS A CONFIRMATORY TOOL IN MISMATCHED GLANZMANN THROMBASTHENIA
- Severe iron-deficiency anemia and recurrent mucosal bleeding revealing Glanzmann thrombasthenia in a young adult: a case report
- PO096 | IDENTIFICATION OF A NEW VARIANT IN THE RASGRP2 GENE IN A CHILD WITH SUSPECTED GLANZMANN THROMBASTHENIA
- Two novel missense variants altering cytosolic phospholipase A2α structural dynamics partially affect platelet signaling pathways associated with bleeding among Glanzmann thrombasthenia patients from two unrelated south Indian families
- CO006 | PREVALENCE OF GASTROINTESTINAL BLEEDING IN GLANZMANN THROMBASTHENIA PATIENTS: RESULTS OF A WORLDWIDE SURVEY
BibTeX
@article{tarawah2026clinical,
title = {{Clinical and economic impact of rFVIIa prophylaxis in Glanzmann thrombasthenia: cohort analysis in Saudi Arabia.}},
author = {Ahmad Tarawah and Sami Albattat and Ayman Shahba and Mohamed Nashat and Sarah Brighton and Emily Back and Sam Bristow and George Morgan},
journal = {Blood vessels, thrombosis \& hemostasis},
year = {2026},
month = sep,
doi = {10.1016/j.bvth.2026.100219},
url = {https://europepmc.org/article/MED/42903760},
note = {CC BY-NC-ND},
}
RIS
TY - JOUR
TI - Clinical and economic impact of rFVIIa prophylaxis in Glanzmann thrombasthenia: cohort analysis in Saudi Arabia.
AU - Ahmad Tarawah
AU - Sami Albattat
AU - Ayman Shahba
AU - Mohamed Nashat
AU - Sarah Brighton
AU - Emily Back
AU - Sam Bristow
AU - George Morgan
PY - 2026
DA - 2026/09/16
JO - Blood vessels, thrombosis & hemostasis
DO - 10.1016/j.bvth.2026.100219
UR - https://europepmc.org/article/MED/42903760
AB - Glanzmann thrombasthenia (GT) is an ultrarare inherited platelet disorder associated with lifelong bleeding tendency and substantial health care burden. Evidence on the clinical and economic impact of prophylaxis in GT remains limited. We conducted a prospective cohort analysis of pediatric patients with severe GT receiving prophylactic recombinant activated factor VII (rFVIIa) at a specialist tertiary center in Saudi Arabia. Annualized clinical outcomes and direct medical costs during prophylaxis were compared with a historical preprophylaxis period of equal duration for each patient. Outcomes included treated bleeding episodes, hospital days, platelet transfusion use, and rFVIIa consumption. Costs were estimated by applying unit costs to captured resource use. Paired nonparametric analyses and deterministic sensitivity analyses were conducted. Eleven patients were included. Median annualized treated bleeding episodes decreased from 10 during the preprophylaxis period to 4 during prophylaxis. Median annualized hospital days decreased from 96 to 24, whereas platelet transfusion use decreased from 63 to 6 units. Although rFVIIa acquisition costs increased during prophylaxis, these were offset by lower hospitalization and platelet transfusion costs, resulting in a median net annual cost impact of -1305 Saudi riyal (SAR). Deterministic sensitivity analyses demonstrated robustness to plausible variation in costing assumptions, with median net annual cost impact ranging from -93 339 SAR to 90 729 SAR. These findings provide the first evidence indicating clinical and economic benefits associated with rFVIIa prophylaxis in GT and support further evaluation of prophylactic treatment strategies in this population.
SN - 2950-3272
ER -