<abstract xmlns="http://www.w3.org/1999/xhtml"><sec><h3>Background</h3><p>Patients with haemophilia who are not adequately treated experience a lifetime burden of joint complications and loss of functional ability due to repeated bleeding episodes caused by low levels of clotting factor VIII or IX in the blood. These complications can significantly impact day-to-day life, including active participation in school and academic study in children with haemophilia (CwH). Treatment with factor replacement therapy can help to prevent this, but access to factor has been challenging in low-resource settings such as Northeast India. This study shows the impact of factor replacement therapy on bleeding episodes, joint complications and school absence among CwH in this setting.</p></sec><sec><h3>Methods</h3><p>A retrospective observational study was undertaken to examine the impact of receiving regular factor replacement therapy (prophylaxis or on demand) on school absences among CwH registered with the haemophilia treatment centre at Assam Medical College and Hospital. Annual bleed rate (ABR), Haemophilia Joint Health Score (HJHS) and Functional Independence Score in Haemophilia (FISH) were also assessed.</p></sec><sec><h3>Results</h3><p>Thirty-eight CwH were eligible for the study; 26 (68.4%) were on prophylaxis therapy and 12 (31.5%) received on-demand therapy. In the year before starting regular treatment, the mean ABR was 37.8 (+20.0), HJHS was 31.1 (+18.1) and mean FISH score was 21.1 (+4.2). At the end of the study period the mean (+SD) ABR in prophylactic therapy was significantly lower at 5.8 (+4.6) (p&lt;0.001) and the HJHS was significantly lower at 4.7 (+4.6). FISH score significantly improved to 27.9 (+3.3) (p&lt;0.001). Prophylaxis showed better (but not significant) results in comparison to on-demand therapy.</p></sec><sec><h3>Conclusions</h3><p>Treatment with factor replacement significantly reduces school absence in CwH and correlates strongly with joint health and functional improvement, with the effect slightly better with prophylaxis than on-demand therapy. Low dose prophylaxis is a good treatment option in low-resource settings, but improvements are also needed in rates of diagnosis.</p></sec></abstract>

BackgroundPatients with haemophilia who are not adequately treated experience a lifetime burden of joint complications and loss of functional ability due to repeated bleeding episodes caused by low levels of clotting factor VIII or IX in the blood. These complications can significantly impact day-to-day life, including active participation in school and academic study in children with haemophilia (CwH). Treatment with factor replacement therapy can help to prevent this, but access to factor has been challenging in low-resource settings such as Northeast India. This study shows the impact of factor replacement therapy on bleeding episodes, joint complications and school absence among CwH in this setting.MethodsA retrospective observational study was undertaken to examine the impact of receiving regular factor replacement therapy (prophylaxis or on demand) on school absences among CwH registered with the haemophilia treatment centre at Assam Medical College and Hospital. Annual bleed rate (ABR), Haemophilia Joint Health Score (HJHS) and Functional Independence Score in Haemophilia (FISH) were also assessed.ResultsThirty-eight CwH were eligible for the study; 26 (68.4%) were on prophylaxis therapy and 12 (31.5%) received on-demand therapy. In the year before starting regular treatment, the mean ABR was 37.8 (+20.0), HJHS was 31.1 (+18.1) and mean FISH score was 21.1 (+4.2). At the end of the study period the mean (+SD) ABR in prophylactic therapy was significantly lower at 5.8 (+4.6) (p<0.001) and the HJHS was significantly lower at 4.7 (+4.6). FISH score significantly improved to 27.9 (+3.3) (p<0.001). Prophylaxis showed better (but not significant) results in comparison to on-demand therapy.ConclusionsTreatment with factor replacement significantly reduces school absence in CwH and correlates strongly with joint health and functional improvement, with the effect slightly better with prophylaxis than on-demand therapy. Low dose prophylaxis is a good treatment option in low-resource settings, but improvements are also needed in rates of diagnosis.

Improvement in school absence after factor replacement in students with haemophilia in Upper Assam, India

The Journal of Haemophilia Practice

This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 3.0 License.

BackgroundPatients with haemophilia who are not adequately treated experience a lifetime burden of joint complications and loss of functional ability due to...

VARIABLE	PROPHYLAXIS MEAN (±SD)	ON-DEMAND MEAN (±SD)
Age at enrolment (years)	13.1(±4.5)	11.7(+4.5)
Duration of prophylaxis (years)	1.4(±0.2)	–
Haemophilia type
Haemophilia A	21	10
Haemophilia B	5	2

	BEFORE ENROLMENT	P-VALUE	AFTER ENROLMENT	P-VALUE
Prophylaxis
ABR	0.514	0.009	0.639	<0.001
HJHS	0.325	0.112	0.156	0.446
FISH	−0.751	<0.001	−0.694	<0.001
On-demand
ABR	0.874	<0.001	0.396	0.203
HJHS	0.690	0.013	0.197	0.539
FISH	−0.798	0.002	−0.580	0.048

VARIABLE	BEFORE ENROLMENT MEAN(±SD)	AFTER ENROLMENT MEAN(±SD)	P-VALUE^{^*}
Number of days absent (prophylaxis)	15(+6)	4(+3)	<0.001
Number of days absent (on-demand)	10(+5)	3(+2)	0.477
Scores during prophylaxis
ABR	37.8(±20.0)	5.8(±4.6)	<0.001
HJHS	31.1(±18.1)	4.7(±4.6)	<0.001
FISH	21.4(±4.2)	27.9(±3.3)	<0.001
Scores during on-demand treatment
ABR	25.5(±19.9)	8.7(±6.2)	0.006
HJHS	13.8(±11.4)	3.1(±4.2)	0.002
FISH	24.6(±4.5)	29.1(±2.6)	<0.001

Improvement in school absence after factor replacement in students with haemophilia in Upper Assam, India

Article Category: Clinical Research

Published Online: Nov 19, 2020

Page range: 136 - 142

DOI: https://doi.org/10.17225/jhp00166

Keywords
Haemophilia, school absence, factor replacement therapy, prophylaxis, on-demand therapy, Northeast India

© 2020 Anupam Dutta et al., published by Sciendo

This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 3.0 License.

Characteristics of participants

Correlation between school absences and ABR, HJHS and FISH during prophylaxis and on-demand treatment

Days of school absence (per month) in the year before and after enrolment with HTC

Improvement in school absence after factor replacement in students with haemophilia in Upper Assam, India

Article Category: Clinical Research

Published Online: Nov 19, 2020

Page range: 136 - 142

DOI: https://doi.org/10.17225/jhp00166

KeywordsHaemophilia, school absence, factor replacement therapy, prophylaxis, on-demand therapy, Northeast India

© 2020 Anupam Dutta et al., published by Sciendo

This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 3.0 License.

Characteristics of participants

Correlation between school absences and ABR, HJHS and FISH during prophylaxis and on-demand treatment

Days of school absence (per month) in the year before and after enrolment with HTC

Keywords
Haemophilia, school absence, factor replacement therapy, prophylaxis, on-demand therapy, Northeast India