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<p>Glanzmann thrombasthenia (GT) is a rare autosomal-recessive platelet function disorder caused by defects in the GPIIb/IIIa receptor, resulting in impaired aggregation despite normal platelet counts. Children most often present with mucocutaneous bleeding of variable severity. Data from the Indian pediatric population remain scarce, and this study provides region-specific clinical and treatment insights. A retrospective review of confirmed pediatric GT cases (2010–2024) was performed. Demographic features, bleeding patterns, laboratory parameters, and treatments were recorded. Subgroup comparisons were conducted for International Society on Thrombosis and Haemostasis Bleeding Assessment Tool (ISTH-BAT) score, Hemoglobin, and Mean Corpuscular Volume (MCV), the most meaningful indicators of bleeding burden and its hematologic consequences. Twenty children were included (55% males, 60% consanguinity). Easy bruising (90%) and epistaxis (55%) were the predominant manifestations, and 45% experienced life-threatening bleeds. Median age at presentation was 10 years, and moderate anemia was common. Most children (70%) responded to tranexamic acid alone, whereas 30% required advanced therapy [platelet transfusion and recombinant factor VIIa (rFVIIa)]. Subgroup analyses showed that females had higher ISTH-BAT scores with lower hemoglobin and MCV; children needing advanced therapy had markedly worse bleeding scores and hematologic indices; early symptom onset (≤ 2 years) was associated with higher ISTH-BAT; and life-threatening bleeds correlated with higher ISTH-BAT and lower hemoglobin and MCV. Children with GT in this region experience substantial bleeding burden and diagnostic delay. Tranexamic acid remains first-line therapy, while rFVIIa is an effective rescue option in severe or refractory bleeding.</p><p><b>Clinical trial registration</b></p><p>Nil.</p>

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Glanzmann Thrombasthenia- Insights From a Tertiary Care Centre in Eastern India

  • 期刊论文

  • 发表日期：

    2026年4月7日

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  • Subham Bhattacharya,
  • Kaustav Ghosh,
  • Tuphan Kanti Dolai

摘要

Glanzmann thrombasthenia (GT) is a rare autosomal-recessive platelet function disorder caused by defects in the GPIIb/IIIa receptor, resulting in impaired aggregation despite normal platelet counts. Children most often present with mucocutaneous bleeding of variable severity. Data from the Indian pediatric population remain scarce, and this study provides region-specific clinical and treatment insights. A retrospective review of confirmed pediatric GT cases (2010–2024) was performed. Demographic features, bleeding patterns, laboratory parameters, and treatments were recorded. Subgroup comparisons were conducted for International Society on Thrombosis and Haemostasis Bleeding Assessment Tool (ISTH-BAT) score, Hemoglobin, and Mean Corpuscular Volume (MCV), the most meaningful indicators of bleeding burden and its hematologic consequences. Twenty children were included (55% males, 60% consanguinity). Easy bruising (90%) and epistaxis (55%) were the predominant manifestations, and 45% experienced life-threatening bleeds. Median age at presentation was 10 years, and moderate anemia was common. Most children (70%) responded to tranexamic acid alone, whereas 30% required advanced therapy [platelet transfusion and recombinant factor VIIa (rFVIIa)]. Subgroup analyses showed that females had higher ISTH-BAT scores with lower hemoglobin and MCV; children needing advanced therapy had markedly worse bleeding scores and hematologic indices; early symptom onset (≤ 2 years) was associated with higher ISTH-BAT; and life-threatening bleeds correlated with higher ISTH-BAT and lower hemoglobin and MCV. Children with GT in this region experience substantial bleeding burden and diagnostic delay. Tranexamic acid remains first-line therapy, while rFVIIa is an effective rescue option in severe or refractory bleeding.

Clinical trial registration

Nil.