الفهرس 
Hemophilia AOnly 14 pages are availabe for public view
 |  | from | 129 |  |  |
| | | from | 129 | | |
Abstract
This study was conducted on patients recruited from the Pediatric Hematology/Oncology Clinic, Children’s Hospital, Ain Shams University, and the Therapeutic Unit of hemophilic patients at Vacsera. It included 60 children and adolescents with hemophilia A aging from 4-16 years old, of various disease severity, they were classified according to the age into 3 age groups, group I (4-7 years): 25 patients (42%), group II (8-12 years): 19 patients (32%) and group III (13-16 years): 16 patients (26%). Evaluation of FVIII–Inhibitory Activity by both a qualitative screening method and quantitative Bethesda method was done. Assessment of QoL for our hemophilic patients was done also using the Haemo-QoL questionnaire.
The clinical data of our patients was shown and It was found that the episode of first bleeding in 82% of the patients was during circumcision, highly significant in group III (100%), the children were reported to have a target joint (83.3, and it was highly significant in group III too (100%). Also limitation of the joint movement was (63.5%), highly significant in group III compared to other age groups. In this study, 58% of the patients suffered from hemarthrosis, with no differences were found between the three age groups. And 39.5% of them suffered from recurrent epixtasis, statistically significant in group II (63.2%). Also 60.2% of our patients had muscle hematoma, which was significant in group III (81.3%). While 68.3% of the patients were reported to have recurrent gum bleeding which was more frequent in age group II, and 42.8% of the patients reported to have bleeding with after dental procedure, was statistically highly significant in age group II (78.9%).
The prevalence of FVIII inhibitors was 10% by qualitative screening method, and 5% by quantitative Bethesda method, which revealed that quantitative Bethesda method is much more accurate than qualitative screening method, which may give false positive results. By the quantitative Bethesda method the prevalence of inhibitors found to be 5%, and it was positive only for three patients, who aging 8 years, 12 years, 7 years respectively, which revealed that the ratio was 4% for group I, 10.5% for group II, 0.0% for group III, and revealed also that the prevalence was prominent in age group II (8-12 years) by quantitative Bethesda method.
The peak titer of the inhibitor was 16 BU and among those three patients positive for FVIII inhibitors, we found that two patients had inhibitors > 5 BU, these patients are considered high responders, which revealed that 66.7% of these patients had been high responders (peak titer > 5 BU), 50% of age group I and 50% of age group II.
The HRQoL of children with hemophilia was assessed with the recently developed disease-specific questionnaire Haemo-QoL which is a self-reported questionnaire for children in the age ranges 4-7 (I: 21 items), 8-12 (II: 64 items), 13-16 years (III: 75 items) as well as for parent rating containing 9-11 subscales (depending on age-group versions). The scores were found to be for all dimensions widely above 50 (in a range of 0-100), which reported that the HRQoL was not satisfactory in our study (as high scores demonstrate bad HRQoL), and it was highly significant in three dimensions (Physical health – Family – treatment) between different age groups, as it was impaired in the dimension of ‘physical health’ for group II and III, and in the dimension of ‘family’ for group III, while group I was highly impaired concerning the ‘treatment’ they received. On the other hand, younger children (group I) showed a higher HRQoL, revealed particularly by the dimension ‘physical health’, and for age group II and III higher HRQoL was revealed particularly by the social dimensions such as ‘perceived support’ and ‘friends’ and also in the dimension of ‘dealing’ which concerns personal adaptation to the disease. The three age groups were impaired in the dimension of ‘feeling’, and in the dimension of ‘sports/school’, and in the dimension of ‘view of themselves’, and also in the dimension of interaction with ‘others’, age group III was found to be impaired also in other 2 dimensions ‘family’, and their look to ‘future’.
Regarding the presence of inhibitors to FVIII either by qualitative screening method or quantitative Bethesda method, it was found to be insignificant for all dimensions of Haemo-QoL questionnaire, which proved that HRQoL not affected in this study by the presence of FVIII inhibitors within the three age groups.
In conclusion, in spite of an increased interest in the assessment of quality of life (QoL) in children, so far no instrument for children with hemophilia is available. Because of the low prevalence of the condition, such an instrument should also be cross-culturally applicable. Also international development of hemophilia specific measures for QoL, both for adults as well as children, are necessary in order to make available methodologically sound assessment instruments that are relevant to patients and assess a major outcome health related QoL.