Development of a patient-reported outcomes tool to monitor changes in joint health and wellbeing for young people with haemophilia B

Figure 1.

Figure 2.

Literature review search strategy Databases searched included Embase 1996 to 2020 Week 50, MEDLINE® 1946 to 16 December 2020, and PsycINFO® 2002 to December Week 1 2020_

STEP	SEARCH TERMS	SEARCH TYPE	HITS
1.	H?emophilia* or Christmas disease or factor IX deficien* or FIX deficien*	All fields	76,045
2.	joint bleed* or microbleed* or asymptomatic bleed* or synovial bleed* or silent bleed* or subclinical bleed* or h?emophilic arthropathy or h?emarthtro* or joint pain	All fields	32,949
3.	symptom* or impact* or daily activit* or QOL or health related quality life or HRQOL or activit* daily living or ADL	All fields	6,918,773
4.	Qualitative or (focus ADJ group)* or interview* or patient report* or self report* or parent report* or caregiver report* or CareRO or observer report* or ObsRO or P?ediatri* or adolescen*	All fields	8,836,985
5.	(1) AND (2) AND (3) AND (4)	-	514
6.	Limited to English language	-	500
7.	Limit to full text	-	198
8.	Limited to Human	-	177
9.	Limit to last 10 years ("2010-Current")	-	147
10.	Duplicates removed	-	122

j_jhp-2024-0014_tab_004

AGE (YEARS)	NUMBER OF CHILDREN WITH MILD, MODERATE, OR SEVERE HAEMOPHILIA
	NONE	ONE	TWO OR MORE
7 or younger	Screenout	Screenout	Screenout
8 to 17	Screenout	Proceed	Proceed
18 or older	Screenout	Screenout	Screenout

Advisory board participant information (N=8)

ROLE	WORK SETTING	EXPERIENCE
Consultant Haematologist (#1) (Adults)	CCC; haemophilia database; HaemTrack	Experience working with young people with haemophilia B that have transitioned into adult services
Consultant Haematologist and Centre Director (Adults and Children) (#2)	CCC	Experience working with adults and children with haemophilia
Haemophilia Nurse Specialist (Children) (#3)	CCC; haemophilia database review group	Significant experience working with children with haemophilia
Haemophilia Nurse Specialist (Children) (#4)	CCC	Significant experience working with people with haemophilia, both adults and children
Clinical Specialist Physiotherapist in Haemophilia (Adults) (#5)	CCC; HCPA	Significant clinical and research experience
Clinical Specialist Physiotherapist in Haemophilia (Children) (#6)	CCC	Significant clinical and research experience
Clinical Specialist Physiotherapist in Haemophilia (Adults) (#7)	CCC	Significant clinical and research experience
Paediatric and Adolescent Rheumatologist and Senior Clinical Lecturer (#8)	Rheumatology clinic for adolescents and university centre for musculoskeletal research	Significant experience researching PRO measures and patient-reported experience measures, working with adolescents with musculoskeletal complaints in their clinical practice

Focus group participant information (N=12)

HEALTHCARE PROFESSIONAL	YPWHB (AGE)	HAEMOPHILIA SEVERITY	PARENT (N)
Group 1
Clinical psychologist	9 years*	Moderate	1
	10 years	Moderate	1
	10 years	Mild	1
	11 years	Mild	1
Group 2
Physiotherapist	14 years	Severe	1
	21 years	Severe