Table 1.
Study characteristics (n = 64)
| Study Characteristics | % | Frequency |
|---|---|---|
| Decade published | ||
| 1990-1999 | 9.0 | 6 |
| 2000-2009 | 16.0 | 10 |
| 2010-2019 | 53.0 | 34 |
| 2020 to present | 22.0 | 14 |
| Location | ||
| United States Only | 73.0 | 47 |
| United States and other country | 14.0 | 9 |
| Location unclear only | 11.0 | 7 |
| United States and location unclear | 2.0 | 1 |
| Funding source | ||
| Public | 31.0 | 20 |
| Private | 13.0 | 8 |
| Public-private collaboration | 14.0 | 9 |
| Not mentioned | 42.0 | 27 |
| Intervention type | ||
| BMT/SCTa only | 91.0 | 58 |
| Gene therapy (GT) only | 5.0 | 3 |
| BMT/SCT/GT | 5.0 | 3 |
| Study design | ||
| Experimental only | 41.0 | 26 |
| Observational only | 38.0 | 24 |
| Qualitative only | 9.0 | 6 |
| Economic evaluation only | 3.0 | 2 |
| Experimental and observational | 3.0 | 2 |
| Observational and qualitative | 6.0 | 4 |
| Population characteristics | ||
| Illness population | ||
| Sickle cell disease (SCD) only | 72.0 | 47 |
| Beta thalassemia (BT) only | 16.0 | 10 |
| SCD and beta thalassemia | 12.0 | 7 |
| Stakeholder population | ||
| People with illness only | 78.0 | 50 |
| Family and/or caregiver | 8.0 | 5 |
| People with illness and family and/or caregiver | 15.0 | 9 |
| Age group | ||
| Pediatric only | 20.0 | 12 |
| Adult only | 23.0 | 15 |
| Pediatric and adult | 52.0 | 34 |
| Not mentioned/unclear | 7.0 | 4 |
BMT, bone marrow transplantation; BT, beta thalassemia; GT, Gene therapy; SCD, sickle cell disease; SCT, stem cell transplant.
a
Bone marrow transplant/stem cell transplant.