Table 3. Summary of Treatment Trials: Key Question 2a
Study, Year (Reference) |
Population, n |
Treatment |
Measure and Results |
Adams, et al., 199125 |
85 Probands and family
members |
500 mL of blood/wk
until serum ferritin level
< 30 µg/L or patient
became anemic |
Cumulative survival, %:
5 y: 87
10 y: 81
20 y: 71
Adjusted relative risk for death:
Cirrhosis: 5.54
Arthritis: 0.24 |
|
Niederau, et al., 199660 |
251 Diagnosed through
routine clinical
practice |
500 mL of blood/1-2 wk
until serum ferritin
levels were normal |
Cumulative survival, %:b
5 y: 93
10 y: 77
20 y: 55
30 y: 20
Select Table 3a for changes in fibrosis stage after iron depletion and Table 3b for details of symptoms. |
Bomford and
Williams, 197659 |
Treated: 85
Controls: 26
Diagnosed through
routine clinical
practice |
600 mL of blood/wk
until hemoglobin value
<100 g/L and serum
iron level <10 µmol/L |
Diabetes, n/n (%):
Improved: 16/56 (29)
Worsened: 7/56 (13)
New cases: 3
Liver histologic features, n/n (%):
Improved: 5/75 (7)
No definite change: 68/75 (91)
Worsened: 2/75 (3)
|
McDonnell et al.,
199955,c |
2851
Population-based
mailing to persons
known to have
hemochromatosis |
Varied |
Some or all of symptoms improved with therapy: 86%.
New symptoms developed despite treatment: 33%
Select Table 3c for details of symptoms. |
Powell, et al.,
200658 |
25
Selected subset of
cases diagnosed
through family
screening or
work-up of
elevated iron
measures |
Unspecified |
Change in fibrosis stage after iron depletion, n/n (%):
Improved: 19/20 (95)
Unchanged (cirrhosis at baseline): 1/20 (5)
Not reported because of high alcohol intake: 5/25 (20) |
a. AD = at diagnosis; ALT = alanine aminotransferase; AST = aspartate aminotransferase; I = improved; U = unchanged; W = worsened.
b. Significantly reduced compared with expected survival in matched population.
c. Compared with National Health and Nutrition Examination Surveys II and III, similar proportion of patients reported arthritis, liver or gallbladder disease, and extreme
fatigue as general population.
Table 3a. Changes in Fibrosis Stage After Iron Depletion (n = 185)
Stage |
I, n | W, n |
U, n |
0 |
0 |
1 | 20 |
1 |
10 |
1 | 21 |
2 |
20 |
0 | 19 |
3 |
12 |
0 | 81 |
Total |
42(23%) |
2(1%) | 141 (76%) |
Return to Table 3
Table 3b. Details of Symptoms from Niederau, et al.,
199660 Study
Sign/Symptom |
AD, % | I, % |
U, % | W, % |
Weakness/lethargy |
80 |
55 | 40 | 6 |
Abdominal pain |
56 |
68 | 29 | 1 |
Arthralgia |
45 |
50 | 30 | 20 |
Elevated AST or ALT level |
81 |
73 | 25 | 2 |
Pigmentation |
68 |
68 | 32 | 0 |
Loss of potency |
40 |
19 | 69 | 12 |
Electrocardiographic changes |
35 |
34 | 61 | 5 |
Diabetes mellitus |
44 |
41 | 53 | 6 |
Impaired glucose tolerance |
15 |
37 | 56 | 7 |
Return to Table 3
Table 3c. Details of Symptoms from McDonnell, et al.,
199955 Study
Sign/Symptom |
All Patients, n(%) | I, n(%)d | W n(%)e |
Extreme fatigue |
1296 (45.5) |
705 (54.4) | 223 (17.2) |
Joint pain |
1241 (43.5) |
115 (9.2) | 422 (34.0) |
Impotence/loss of libido |
735 (25.8) |
93 (12.7) | 204 (27.8) |
Skin bronzing |
733 (25.7) |
431 (58.8) | 30 (4.1) |
Heart fluttering |
679 (23.8) |
42 (6.2) | 69 (10.1) |
Depression |
592 (20.8) |
242 (40.8) | 61 (10.3) |
Abdominal pain |
578 (20.3) |
129 (22.3) | 69 (11.9) |
d. Improved with treatment.
e. Worsened despite treatment.
Return to Table 3
Return to Document
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