Comparison of Pulmonary Function, Exercise Capacity, and Physical Activity Level Between Children With Hemophilia and Healthy Controls

NCT06636565 | Completed

• 1 other identifier: FTREK24/65
• Study Type: observational
• Target: 34
• Locations: 1 country
• Sites: 1

Brief Summary

Hemophilia is a rare X-linked congenital bleeding disorder characterized by deficiency of clotting factor VIII (hemophilia A) or deficiency of factor IX (hemophilia B) with complex diagnosis and management. Participation in physical activity is still limited in children with hemophilia, probably due to protective attitudes of families/patients and avoidance of activity, and possibly also as a result of chronic pain. Exercise capacity has been identified as a protective factor against joint problems in hemophilia. Aerobic fitness is associated with better pulmonary function in children. Chronic pain and decreased range of motion due to recurrent bleeding in joints and muscles in hemophilia may indirectly affect posture and respiratory mechanics, leading to impaired pulmonary function. This study aims to compare pulmonary function, exercise capacity, posture, and physical activity level between children with hemophilia and healthy controls and to investigate the relationship between these parameters.

Conditions

Hemophilia; Healthy Children

Keywords

Hemophilia; Exercise capacity; Pulmonary function

Outcome Measures

Primary Outcomes (9)

• Forced vital capacity

  Pulmonary function test using a spirometer will be performed. Forced vital capacity will be recorded.

  1st day

• Forced expiratory volume in one second

  Pulmonary function test using a spirometer will be performed. Forced expiratory volume in one second will be recorded.

  1st day

• Forced expiratory volume in one second/forced vital capacity ratio

  Pulmonary function test using a spirometer will be performed. Forced expiratory volume in one second/forced vital capacity ratio will be recorded.

  1st day

• Peak expiratory flow

  Pulmonary function test using a spirometer will be performed. Peak expiratory flow will be recorded. will be recorded.

  1st day

• Forced mid-expiratory flow (FEF25-75)

  Pulmonary function test using a spirometer will be performed. Forced mid-expiratory flow (FEF25-75) will be recorded. will be recorded.

  1st day

• Hemophilia Joint Health Score

  Hemophilia Joint Health Score is a scoring used by physiotherapists in hemophilia treatment centers to detect early hemophilic arthropathy symptoms and to monitor joint health. HJHS consists of edema (0-3), duration of edema (0-1), muscle atrophy (0-2), crepitus during movement (0-2), loss of flexion (0-3), loss of extension (0-3), joint pain (0-2) and evaluation of muscle strength of knees, ankles and elbows (0-4) and global gait score (0-4). The maximum score is 124, with higher scores indicating worse joint health.

  1st day

• Corbin Posture Rating Scale

  Corbin Posture Rating Scale and observational posture analysis. It is an assessment consisting of lateral and posterior posture analysis. '0' is excellent, 12 and above indicates poor posture.

  1st day

• Exercise capacity

  To assess exercise capacity, a symptom-limited cardiopulmonary exercise test will be performed on a bicycle ergometer using Godfrey protocol. Cardiopulmonary responses and effort perceptions will be recorded. In Godfrey protocol, workload increases are adjusted according to height: workload is increased by 10 W/min for \< 125 cm, by 15 W/min for 125-150 cm and by 20 W/min for \>150 cm.

  1st day

• Physical activity level

  The Physical Activity Questionnaire for Children (PAQ-C) and the Physical Activity Questionnaire for Adolescents (PAQ-A) will be used to assess physical activity levels. The Turkish versions of these assessment tools have been shown to be valid and reliable.

  1st day

Study Arms (2)

Hemophilia

Children with hemophilia

Other: No intervention

Healthy children

Healthy children

Other: No intervention

Interventions

No intervention OTHER

No intervention

Healthy children; Hemophilia

Eligibility Criteria

• Age: 8 Years - 18 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Probability Sample

Study Population

Children with hemophilia and their healthy peers

You may qualify if:

• Having been diagnosed with hemophilia and being clinically stable,
• Being between the ages of 8-18,
• Being on prophylaxis treatment (a routine treatment),
• Being 125 cm or taller,
• Being inhibitor negative at the time of enrollment,
• To be able to cooperate with the tests to be performed,
• Being willing to participate in the study.

You may not qualify if:

• Being inhibitor positive at the time of enrollment,
• Having a history of acute joint bleeding,
• Being unable to bear weight on the extremities,
• Having a history of acute intramuscular bleeding,
• Having undergone radionuclide synovectomy,
• Having a history of surgery on any of the lower extremity joints,
• Having a severe cardiovascular, orthopedic or neurological problem that may affect the assessments.
• Control group
• Not having any known internal or chronic disease
• Being male between the ages of 8-18
• Having full range of motion in the upper extremity
• Having full range of motion in the lower extremity
• Not having any history of injury or surgery in the lower extremity
• Being 125 cm or taller
• Being willing to participate in the study

Sponsors & Collaborators

• Hacettepe University: lead

Study Sites (1)

Hacettepe University Faculty of Physical Therapy and Rehabilitation

Ankara, Ankara, 06100, Turkey (Türkiye)

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Aslihan Cakmak-Onal, PhD, PT

  Hacettepe University

  PRINCIPAL INVESTIGATOR

• Ebru Calik-Kutukcu, PhD, PT

  Hacettepe University

  STUDY DIRECTOR

• Hande Guney-Deniz, PhD, PT

  Hacettepe University

  STUDY CHAIR

• Ş. Selin Aytac-Eyupoglu, MD

  Hacettepe University

  STUDY CHAIR

• Firat Tan, MSc, PT

  Ataturk University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: October 7, 2024
• First Posted: October 10, 2024
• Study Start: July 25, 2024
• Primary Completion: July 25, 2025
• Study Completion: July 25, 2025
• Last Updated: September 10, 2025

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

TU (1)