Article summary
What’s known on this subject
What this study adds
Introduction
Methods
Facebook recruitment campaign
Study participants
Survey instrument
Statistical analysis
Results
Survey Respondent Characteristics | n (%) |
---|---|
Survey respondent’s age (years)
| |
Mean (SD) | 39.4 (8.73) |
Child’s sex
| |
Female | 63 (45.3%) |
Male | 76 (54.7%) |
Relationship to child with PH
| |
Father | 10 (7.2%) |
Guardian | 3 (2.2%) |
Mother | 124 (89.2%) |
Primary caregiver | 2 (1.4%) |
Respondent's educational attainment
| |
High school graduate (includes equivalency, i.e., GED) | 9 (6.5%) |
Some college, no degree | 26 (18.7%) |
Associate’s degree | 18 (12.9%) |
Bachelor’s degree | 45 (32.4%) |
Graduate or professional degree | 39 (28.1%) |
Prefer not to say | 2 (1.4%) |
Employment status
| |
Employed | 89 (64.0%) |
Homemaker | 34 (24.5%) |
Unemployed | 14 (10.1%) |
Prefer not to say | 2 (1.4%) |
Household income
| |
< $35,000 | 16 (11.5%) |
$35,000 to $74,999 | 37 (26.6%) |
> $75,000 | 76 (54.7%) |
Prefer not to say | 10 (7.2%) |
Home ownership
| |
Own (or paying mortgage) | 108 (77.7%) |
Rent | 30 (21.6%) |
Prefer not to say | 1 (0.7%) |
How long lived in current residence
| |
Less than 1 year | 11 (7.9%) |
1 to 2 years | 16 (11.5%) |
2 to 3 years | 22 (15.8%) |
3 to 5 years | 20 (14.4%) |
5 or more years | 69 (49.6%) |
Prefer not to say | 1 (0.7%) |
N (%)
| |
---|---|
Parent/Guardian Reported Birth Defect
| |
Yes | 67 (48.2) |
No | 72 (51.8%) |
Total # of Birth Defects Reported
| 119 |
# of Birth Defects Reported per Child (mean, SD)
| 1.13 (1.58) |
Birth defects reported by respondentsa
| |
Left Heart Obstructive Lesions
| |
Coarctation of the Aorta | 4 (6%) |
Aortic Valvar Stenosis (AS) | 1 (1.5%) |
Interrupted Aortic Arch / VSD | 2 (3%) |
Shone's Complex | 2 (3%) |
Conotruncal Abnormalities
| |
Pulmonary Atresia (PA) | 1 (1.5%) |
Pulmonary Valvar Stenosis (PS) | 1 (1.5%) |
Taussig-Bing Variant DORV | 1 (1.5%) |
Tetralogy of Fallot | 3 (4.5%) |
Transposition of the Great Arteries | 10 (14.9%) |
Absent left pulmonary artery | 1 (1.5%) |
Pulmonary Venous Abnormalities
| |
Scimitar syndrome | 1 (1.5%) |
Partial Anomalous Pulmonary Venous Return (PAPVR) | 2 (3%) |
Total Anomalous Pulmonary Venous Return (TAPVR) | 1 (1.5%) |
Systemic Venous Abnormalities
| |
Persistent left superior vena cava | 1 (1.5%) |
Septation Defects and Extracardiac Shunts
| |
Atrial Septal Defect (ASD) | 28 (41.8%) |
Atrioventricular Septal Defect (or AV Canal Defect) | 8 (11.9%) |
Patent Ductus Arteriosus (PDA) | 21 (31.3%) |
Ventricular Septal Defect (VSD) | 20 (29.9%) |
Patent Foramen Ovale (PFO) | 3 (4.5%) |
Single Ventricle Physiology
| |
Hypoplastic Left Heart Syndrome | 2 (3%) |
Single Ventricle Anomalies NOS | 2 (3%) |
Airway/Respiratory Abnormalities/Other
| |
Bronchopulmonary Dysplasia (BPD) | 2 (3%) |
Bronchomalacia | 1 (1.5%) |
Laryngomalacia | 1 (1.5%) |
Child's Characteristics | n (%) |
---|---|
Child’s race/ethnicity
| |
White/Caucasian | 114 (82.0%) |
Other | 25 (18.0%) |
Hispanic
| |
Yes | 6 (4.3%) |
No | 133 (95.7%) |
Citizenship status
| |
U.S. Resident | 119 (85.6%) |
Outside U.S | 15 (10.8%) |
Prefer not to say | 5 (3.6%) |
Gestational age at birth
| |
Less than 24 weeks | 6 (4.3%) |
More than 36 weeks | 87 (62.6%) |
24 to 28 weeks | 12 (8.6%) |
29 to 32 weeks | 4 (2.9%) |
33 to 36 weeks | 21 (15.1%) |
I don’t know | 9 (6.5%) |
Age at PH diagnosis
| |
First 30 days | 32 (23.0%) |
1–12 months | 37 (26.6%) |
1–4 years | 40 (28.8%) |
5–10 years | 23 (16.5%) |
11–15 years | 7 (5.0%) |
Current age
| |
< 1 year | 13 (9.4%) |
1–5 years | 45 (32.4%) |
6–10 years | 36 (25.9%) |
11–15 years | 28 (20.1%) |
16–17 years | 15 (10.8%) |
> = 18 years | 2 (1.4%) |
Down Syndrome
| |
Yes | 12 (8.6%) |
No | 126 (90.6%) |
Prefer not to say | 1 (0.7%) |
Congenital heart defect
| |
Yes | 67 (48.2%) |
No | 72 (51.8%) |
Health insurance
| |
Yes | 131 (94.2%) |
No | 6 (4.3%) |
Prefer not to say | 2 (1.4%) |
Offered genetic testing at diagnosis
| |
Yes | 81 (58.3%) |
No | 54 (38.8%) |
I don’t know | 3 (2.2%) |
Prefer not to say | 1 (0.7%) |
Genetic testing performed
| |
Yes | 76 (93.8%) |
No | 5 (6.2%) |
Genetic counselor discussed results
| |
Yes | 63 (82.9%) |
No | 9 (11.8%) |
I don’t know | 3 (3.9%) |
Prefer not to say | 1 (1.3%) |
Medication | n (%) |
---|---|
Number of medications used
| |
0 | 14 (10.1%) |
1 | 25 (18.0%) |
2 | 28 (20.1%) |
3 | 56 (40.3%) |
4 | 16 (11.5%) |
Mean (SD) | 2.25 (1.18) |
Oxygen usage
| |
Yes | 84 (60.4%) |
No | 55 (39.6%) |
When oxygen is used
| |
At nighttime only | 36 (38.3%) |
All the time (continuously) | 27 (28.7%) |
As needed | 12 (12.8%) |
When sick | 7 (7.4%) |
At high altitude | 5 (5.3%) |
Only when they are under exertion (moving) | 4 (4.3%) |
Eating | 2 (2.1%) |
When not in School | 1 (1.1%) |
Aspirin
| |
Yes | 33 (23.7%) |
No | 105 (75.5%) |
I don’t know | 1 (0.7%) |
Discussion
Parenteral Prostacyclin | Phosphodiesterase Inhibitors (PDE 5) | Endothelin Receptor Antagonists (ERAs) | Oral Prostacyclin Receptor Agonist | Inhaled Prostacyclin | Soluble Guanylate Cyclase | ||||
---|---|---|---|---|---|---|---|---|---|
Treprostinil (Remodulin) | Tadalafil (Adcirca) | Sildenafil (Revatio) | Ambrisentan (Letairis) | Bosentan (Tracleer) | Macitentan (Opsumit) | Selexipag (Uptravi) | Treprostinil (Tyvaso) | Riociguat (Adempas) | |
Number of children taking Medication
| 43 | 70 | 49 | 56 | 19 | 12 | 26 | 2 | 2 |
Total # of Side Effects Reported
| 190 | 183 | 74 | 30 | 5 | 8 | 74 | 2 | 3 |
Average # of Side Effects per Child (mean, SD)
| 4.40 (2.44) | 2.40 (1.20) | 1.61 (0.86) | 0.53 (0.90) | 0.26 (0.56) | 0.67 (1.50) | 2.85 (2.13) | 1.0 (0.0) | 1.50 (0.71) |
Parent/Guardian Reported ≥ 1 Side Effect
| |||||||||
Yes | 42 (97.7%) | 62 (88.6%) | 36 (73.5%) | 19 (33.9%) | 4 (21.1%) | 3 (25.0%) | 21 (80.8%) | 1 (50.0%) | 1 (50.0%) |
No | 1 (2.3%) | 8 (11.4%) | 13 (26.5%) | 37 (66.1%) | 15 (78.9%) | 9 (75.0%) | 5 (19.2%) | 1 (50.0%) | 1 (50.0%) |
Side Effects Reported
| |||||||||
Flushing of the skin | 31 (72.1%) | 35 (50.0%) | 26 (53.1%) | - | - | - | 13 (50.0%) | 1 (50.0%) | - |
Difficulty sleeping | 2 (4.7%) | 16 (22.9%) | 8 (16.3%) | - | - | - | - | - | - |
Headache/Dizziness | 11 (25.6%) | 28 (40.0%) | 6 (12.2%) | 4 (7.1%) | 1 (5.3%) | 3 (25.0%) | 12 (46.1%) | - | - |
Low blood pressure | 1 (2.3%) | - | 1 (2.0%) | - | - | - | 1 (3.8%) | - | - |
Nasal congestion | - | 37 (52.8%) | 10 (20.4%) | - | - | - | - | - | - |
Nausea | - | 24 (34.3%) | 8 (16.3%) | - | - | - | 12 (46.1%) | - | 1 (50.0%) |
Diarrhea | 15 (34.9%) | - | - | - | - | - | 10 (38.5%) | - | 1 (50.0%) |
Vomiting | 1 (2.3%) | - | - | - | - | - | 3 (11.5%) | - | - |
Heartburn | - | 24 (34.3%) | 8 (16.3%) | - | - | - | - | - | 1 (50.0%) |
Nose bleeds | - | 19 (27.1%) | 7 (14.3%) | - | - | - | - | - | - |
Chest pain | - | - | - | 7 (12.5%) | - | - | - | - | - |
Fever | 4 (9.3%) | - | - | 1 (1.8%) | - | - | - | - | - |
Irregular heart beat | - | - | - | 1 (1.8%) | 1 (5.3%) | - | - | - | - |
Itching | 19 (44.2%) | - | - | 3 (5.4%) | - | 1 (8.3%) | - | - | - |
Joint/Muscle Pain | 20 (46.5%) | - | - | 10 (17.9%) | 2 (10.5%) | 2 (16.6%) | 12 (46.1%) | - | - |
Sore throat | - | - | - | 1 (1.8%) | - | 1 (8.3%) | - | 1 (50.0%) | - |
Swelling | 27 (62.7%) | - | - | 3 (5.4%) | 1 (5.3%) | - | - | - | - |
Fainting | - | - | - | - | - | 1 (8.3%) | - | - | - |
Fatigue | - | - | - | - | - | - | 1 (3.8%) | - | - |
Jaw pain | - | - | - | - | - | - | 3 (11.5%) | - | - |
Decreased Appetite | 1 (2.3%) | - | - | - | - | - | 5 (19.2%) | - | - |
Low red blood cell count | - | - | - | - | - | - | 2 (7.7%) | - | - |
Rash | 16 (37.2%) | - | - | - | - | - | - | - | - |
Scars | 1 (2.3%) | - | - | - | - | - | - | - | - |
Tenderness around infusion site | 41 (95.3%) | - | - | - | - | - | - | - | - |
Care Provider Characteristics
| n (%) |
---|---|
Where PH care is received
| |
Local pediatrician’s office | 2 (1.4%) |
Specialty Children’s Hospital | 133 (95.7%) |
There is no PH care where we live | 4 (2.9%) |
Type of PH provider
| |
Pediatric cardiologist & pediatric pulmonologist | 61 (43.9%) |
Pediatric Cardiologist | 56 (40.3%) |
Pediatric Pulmonologist | 10 (7.2%) |
PH Specialist | 10 (7.2%) |
Primary care physician (Local Pediatrician) | 2 (1.4%) |
Distance to primary PH provider
| |
Less than 5 miles | 4 (2.9%) |
6–10 miles | 8 (5.8%) |
11–20 miles | 13 (9.4%) |
More than 20 miles | 113 (81.3%) |
Prefer not to say | 1 (0.7%) |
Travel time to PH provider
| |
Less than 10 min | 3 (2.2%) |
10–20 min | 5 (3.6%) |
21–30 min | 11 (7.9%) |
30–45 min | 15 (10.8%) |
46–60 min | 9 (6.5%) |
61–90 min | 21 (15.1%) |
More than 90 min | 74 (53.2%) |
Prefer not to say | 1 (0.7%) |
Had to move residence to be closer to PH care provider
| |
Yes | 12 (8.6%) |
No | 127 (91.4%) |