Neutropenia in previously healthy children in Kuwait is caused by demonstrable infections in 55% of cases. Majority of children will recover their ANC completely within 1 month without significant infectious complications.

Although not regularly transfused, patients with non-transfusion-dependent thalassemia (NTDT) are prone to iron overload and its complications. Their molecular, phenotypical and laboratory characteristics vary in different populations and there is a need to document local prevailing patterns. We have reviewed the records of our patients with NTDT in Kuwait and documented their clinical and molecular characteristics in addition to iron status [serum ferritin and liver magnetic resonance imaging (MRI) T2*], management and complications. There were 41 patients, made up of 20 with β-thalassemia intermedia (β-TI), 18 with Hb H (β4) disease and three with Hb E (HBB: c.79G > A)-β-thalassemia (Hb E-β-thal); their ages ranged from 3 to 36 years (mean 12.5 ± 7.7). While 18 (43.9%) had been transfused at least once, only three (7.3%) had been transfused on multiple occasions. Three patients had serum ferritin >500 ng/mL; while four of 38 had mild or moderate liver iron overload. Seven (35.0%) of the β-TI patients were managed with hydroxyurea (HU) with good response. Other complications included five patients with gallstones and one each of hypothyroidism and moyamoya. The most common mutations among the β-TI patients were IVS-II-1 (G > A) and IVS-I-6 (T > C), while among the Hb H patients, the Saudi α2-globin gene polyadenylation (polyA) (AATAAA > AATAAG) mutation was responsible for all cases either as homozygotes (61.1%) or compound heterozygotes with the α-thal-2 (-α(3.7)) allele (33.3%). Although the pattern of NTDT in Kuwaiti patients is generally mild, there is a need to follow them to adulthood as the complications are cumulative and more prevalent in this group.

IntroductionHaemophilia (A, B) is an uncommon inherited disorder worldwide. This study is a review of paediatric haemophilia in Kuwait.MethodThis was a cross-sectional study involving all the haemophilia (A, B) patients currently followed in the paediatric haematology clinics in Mubarak, Amiri and Adan hospitals in Kuwait. Informed consent was obtained as appropriate. Patients’ initial presentation, clinical findings, family history, laboratory data, clinical course, management and complications were documented.ResultThere were 30 patients – 20 haemophilia A and 10 haemophilia B. Of the former, 10 (50%) presented before 6 months of age, 7 (35%) between 6 months to 2 years, 3 (15%) between 2 – 6 years. Five (50%) of haemophilia B presented between 6 months and 2 years, two (20%) between the age 2 – 6 years, two patients after 6 years and one at birth.Nine (45%) of the haemophilia A patients presented with prolonged bleeding, 3 (15%) with mucous membrane bleeding and 3 with hemarthrosis. Two had spontaneous CNS bleeding, and one with intramuscular bleeding.Five (50%) haemophilia B patients presented with mucous membrane bleeding; 30% were diagnosed during routine screening. The rest one with hemarthrosis and one patient with prolonged bleeding after cut wound.There was a positive family history in 13 (65%) patients with haemophilia A and in 80% of haemophilia B. Among the haemophilia A patients, factor deficiency was severe in (75%), moderate in 3 patients and mild in 2 patients. In haemophilia B, 60% were moderate while 40% were severe.Most of the severe patients are on prophylactic treatment with no more evidence of target joints or severe bleeding. Only 3 patients developed high titer inhibitors for factor VIII. They are treated with bypassing agents. None of our patients developed viral infections from the plasma-derived product.ConclusionMost paediatric haemophilia patients in Kuwait have haemophilia A, which presents commonly with prolonged bleeding while haemophilia B (seen in 30%) is more likely to have mucous membrane bleeding.