Introduction
Methods
Phase I (generation of issues)
Literature review
Interviews of patients and physicians specialized in treatment of PNH/AA
Phase II (rating of issues)
Review and rating of issues
Generation of items
Comparison with EORTC QLQ-C30
Results
Literature review
QoL in AA patients
Author | Year | Number of patients evaluated; form of treatment (SCT/IST) | Country of origin | QoL instrument used |
---|---|---|---|---|
Scheinberg [21] | 2011 |
N = 120; IST | USA | Surrogate parameters (blood counts, survival) |
2011 |
N = 49 (adult survivors of pediatric bone marrow transplantation); SCT | USA | SF36; SCL90-R; FRI; SSQSR; NBRS; MMQ | |
Gupta [36] | 2010 |
N = 1307; SCT | Multinational | Surrogate parameters (blood counts, GvHD, survival) |
Resncik [35] | 2006 |
N = 13; SCT | Israel | Surrogate parameters (OS, GvHD, Karnofsky score) |
Viollier [24] | 2005 |
N = 207; SCT and IST | Switzerland | Q-TWiST |
2002 (1991) |
N = 84 | Germany | Surrogate parameters (blood counts, evolution of clonal/malignant disease, CSA side effects) | |
Matsuda [34] | 2002 |
N = 10; SCT | Japan | Surrogate parameters (hemoglobin) |
Goerner [33] | 2002 |
N = 405; SCT | USA | Surrogate parameters (GvHD, Infections, Karnofsky score and others) |
Marsh [32] | 1999 |
N = 115; IST | Multinational | Surrogate parameters (blood counts, time being free of transfusions, OS) |
Deeg [45] | 1998 |
N = 212; SCT | USA | EORTC QLQ-C30 (plus self-developed BMT module); DBMT; POMS; WHPQ |
Passweg [19] | 1997 |
N = 1305; SCT | Multinational | Surrogate parameters (blood counts, GvHD, survival) |
Andrykowski [46] | 1995 |
N = 1 (28)**; SCT (** study comprised of 28 BMT patients including one with aplastic anemia) | USA | POMS, FLIC, PAIS, SIP |
Novitzky [31] | 1992 |
N = 26; IST | South Africa | Surrogate parameters (return to work, blood counts) |
Najean [23] | 1990 |
N = 156; failed SCT, IST (+ androgens) | France | Surrogate parameters (blood counts, occupation, marriage, pregnancies) |
De Planque [22] | 1989 |
N = 468; IST | Multinational | Surrogate parameters (blood counts, survival, clonal evolution) |
Hinterberger [30] | 1987 |
N = 23; SCT | Austria | Surrogate parameters (blood counts, GvHD) |
Bayever [29] | 1984 |
N = 57; SCT, IST (pediatric patients up to the age of 25) | USA | Surrogate parameters (Karnofsky score, GvHD) |
QoL in PNH patients
Author | Year | Number of patients evaluated; form of treatment (SCT/eculizumab/other) | Country of origin | QoL instrument used |
---|---|---|---|---|
Matos-Fernandez [39] | 2009 |
N = 117; SCT (review of SCT in PNH) | Multinational | Surrogate parameters (GvHD, survival) |
Brodsky [12] | 2008 |
N = 93; eculizumab (vs placebo) | Multinational | EORTC QLQ-C30, FACIT |
Meyers [14] | 2007 |
N = 29; other | Multinational | EORTC QLQ-C30, FACIT plus self-reported symptoms |
Hillmen [11] | 2006 |
N = 87; eculizumab (vs placebo) | Multinational | EORTC QLQ-C30, FACIT |
Hill [37] | 2005 |
N = 11 | UK | EORTC QLQ-C30 |
Raiola [38] | 2000 |
N = 7; SCT | Italia | Surrogate parameters (Blood counts, GvHD) |
Patient and physician interviews
Patient and physician samples
Phase I (n = 19) | Phase II (n = 30) | |
---|---|---|
Sex | ||
Female | 15 (79%) | 15 (50%) |
Male | 4 (21%) | 15 (50%) |
Diseasea
| ||
PNH | 4 (21%) | 10 (33%) |
AA | 9 (47%) | 10 (33%) |
AA/PNH | 6 (32%) | 10 (33%) |
Previous therapiesa
| ||
BMT | 3 (16%) | 2 (7%) |
CSA | 13 (68%) | 20 (67%) |
ATG | 13 (68%) | 17 (57%) |
eculizumab | 6 (32%) | 15 (53%) |
Age at diagnosis (in years)a
| ||
Median; mean; range | 29.1; 30.7; 17.0–50.3 | 31.3; 43.2; 13.9–60.3 |
Age at the time of interview (in years) | ||
Median; mean; range | 42.1; 40.7; 25.1–61.3 | 43.7; 43.3; 18.9–73.5 |
Time from first symptoms until first physician contact (in months)a
| ||
Median; mean; range | 0; 9; 0–56 | 0; 3; 0–24 |
Time from first physician contact to final diagnosis (in months)a
| ||
Median; mean; range | 5; 14; 0–56 | 2; 6; 0–37 |
QoL issues
Relevance of issues
Differences between AA and PNH patients
Comparison with the EORTC QLQ-C30 issues
EORTC QLQ-C30 items | Patient rating (mean; median) |
---|---|
Mentioned and rated important | |
1. Do you have any trouble doing strenuous activities, like carrying a heavy shopping bag or a suitcase? | 3.2; 3.0 |
2. Do you have any trouble taking a long walk? | 2.6; 3.0 |
3. Do you need to stay in bed or a chair during the day? | 2.3; 2.0 |
4. Were you limited in doing either your work or other daily activities? | 2.4; 2.0 |
5. Were you limited in pursuing your hobbies or other leisure time activities? | 2.4; 2.0 |
6. Were you short of breath? | 2.9; 3.0 |
7. Did you need to rest? | 3.3; 4.0 |
8. Have you had pain? | 2.5; 3.0 |
9. Did pain interfere with your daily activities? | 2.3; 3.0 |
10. Have you had trouble sleeping? | 2.2; 2.0 |
11. Have you felt weak? | 2.6; 3.0 |
12. Were you tired? | 3.1; 3.0 |
13. Have you had difficulty in concentrating on things, like reading a newspaper or watching television? | 2.4; 2.0 |
14. Did you feel tense? | 2.3; 2.0 |
15. Did you worry? | 2.7; 3.0 |
16. Did you feel depressed? | 2.4; 2.0 |
Mentioned and rated unimportant | |
1. Do you have any trouble taking a short walk outside of the house? | 1.8; 1.0 |
2. Do you need help with eating, dressing, washing yourself or using the toilet? | 1.2; 1.0 |
3. Have you felt nauseated? | 1.6; 1.0 |
4. Have you had diarrhea? | 1.5; 1.0 |
5. Has your physical condition or medical treatment caused you financial difficulties? | 1.9; 1.0 |
6. Has your physical condition or medical treatment interfered with your social activities? | 1.7; 1.0 |
7. Has your physical condition or medical treatment interfered with your family life? | 1.7; 1.0 |
Not mentioned | |
1. Have you lacked appetite? | n.a. |
2. Have you vomited? | n.a. |
3. Have you been constipated? | n.a. |
4. Did you feel tense? | n.a. |
5. Have you had difficulty remembering things? | n.a. |