Table 3.
Specific outcomes measures being used in HD pharmacological trials
| Outcomes Measure | Number of studies using measure(s) |
|---|---|
| CLINICIAN REPORTED OUTCOMES (ClinRO) | |
| Motor Functioning | |
| UHDRS Motor Exam (18) | 61* |
| Abnormal Involuntary Movement Scale (AIMS) (19) | 17 |
| Marsden & Quinn Chorea Severity Scale (20) | 6 |
| Quantitative Neurologic Examination (32) | 3 |
| HD Motor Rating Scale (91); Rockland-Simpson Dyskinesia Rating Scale (92) | 1 |
| Cognitive Functioning | |
| UHDRS Cognitive Exam (18) | |
| Verbal Fluency (22) | 46 |
| Stroop (23) | 43 |
| Symbol Digit Modalities Test (24) | 38 |
| Mini-Mental State Exam (93) | 21 |
| Trailmaking Test (67) | 9 |
| WAIS Digit Span (94, 95) | 5 |
| Benton Visual Retention Test (96); Buschke Selective Reminding Test (97) | 4 |
| AD Assessment Scale-cognitive(98); Raven’s Progressive Matrices Test (99); WAIS Digit Symbol (94, 95) |
3 |
| Hopkins Verbal Learning Test (100); Wechsler Memory Scales (101, 102); WAIS Block Design (94, 95); WAIS Arithmetic (94,95); WAIS FSIQ (94,95); RBANS (103); |
2 |
| Brief Test of Attention (104); CVLT (105); CANTAB (106); CERAD Verbal Learning Test (107); Design Fluency Test (108); Dichotomous Listening Test (109); Digit Ordering Test (110); Go/No |
|
| Go | |
| Test (111); Kohs Cubes Test (112); Luria Nebraska mental rotation item (113); PPVT (114); Recurring Figures Test (115); Road Map Test (116); Ruff Figural Fluency Test (117); Syndrom |
1 |
| Kurz | |
| Test (118); Visual Form Discrimination (119); Washington Square Picture Memory Test (120); Wisconsin Card Sorting Test (121); WAIS Letter Number Sequencing (94, 95) |
|
| Functional Limitations | |
| UHDRS Total Functional Capacity Scale (18) | 48 |
| UHDRS Functional Assessment Scale (18) | 33 |
| UHDRS Independence Scale (18) | 30 |
| HD Activities of Daily Living (ADL) Scale (32) | 2 |
| Emotional/Behavioral Functioning | |
| UHDRS Behavioral Exam (18) | 36 |
| Hamilton Depression Rating Scale (Ham-D) (33, 34) | 8 |
| Brief Psychiatric Rating Scale (122) | 7 |
| Other Assessments | |
| Clinical Global Impression Scale (19) | 13 |
| Clinician Interview Based Impression of Change plus Caregiver (123); Barthel Index(124**) | 1 |
| Study specific | 1 |
| PATIENT REPORTED OUTCOMES (PROs) | |
| Beck Depression Inventory (BDI-II) (42) | 6 |
| Epworth Sleepiness Scale (43) | 3 |
| HADS (44) | 2 |
| NIMH’s Self-Rating Score; Connors’ Adult ADHD Rating Scale (125); SIP (126); SCL-90-R (127) | 1 |
| Study specific | 3 |
| OBSERVER REPORTED OUTCOMES (ObsRO) | |
| Neuropsychiatric Inventory (128) | 2 |
| HD-ADL Scale (32); Activities of Daily Living Scale (129); Cognitive Behavior Rating Scale (130) | 1 |
| OTHER OUTCOME MEASURES | 46 |
| Biomarkers (serum, plasma, CSF levels); Physiology; Neuroimaging |
*
Five of these studies only used a single item (n =1 study used the Luria Hand Position Item and n=4 studies used the Maximal Chorea Item)
**
The study did not specify if the Barthel Index was administered as a ClinRO or a PRO