Impact of strict IGF1 control on quality-of-life scores in patients with acromegaly
ObjectiveExamine, in a real-world setting, whether strict normalization of modestly elevated insulin-like growth factor 1 (IGF1) results in clinical and health-related quality of life benefits in patients with acromegaly using an open-label, non-randomized, 6-month prospective interventional study.M...
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Frontiers Media S.A.
2025-01-01
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| Series: | Frontiers in Endocrinology |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fendo.2025.1516899/full |
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| author | Chaitanya Gandhi Marie-Claire Denis Daniel Holmes Juan Rivera Stan van Uum Shereen Ezzat Constance Chik |
| author_facet | Chaitanya Gandhi Marie-Claire Denis Daniel Holmes Juan Rivera Stan van Uum Shereen Ezzat Constance Chik |
| author_sort | Chaitanya Gandhi |
| collection | DOAJ |
| description | ObjectiveExamine, in a real-world setting, whether strict normalization of modestly elevated insulin-like growth factor 1 (IGF1) results in clinical and health-related quality of life benefits in patients with acromegaly using an open-label, non-randomized, 6-month prospective interventional study.MethodsIn patients with acromegaly and modest IGF1 elevation, strict IGF1 control was achieved by addition or dose escalation of pegvisomant. Clinical and biochemical parameters were assessed at baseline, 1 and 3 months for pegvisomant dose titration, and at 6 months. The Patient-Assessed Acromegaly Symptom Questionnaire (PASQ), the Acromegaly Quality of Life questionnaire (AcroQoL) and the Acromegaly Disease Activity Tool (ACRODAT®) were completed at baseline and at 6 months.ResultsTen patients (8 males) with mean age of 50.7 years participated in the study. All patients had a macroadenoma and nine had prior transsphenoidal surgeries. At time of screening, six patients were on a somatostatin analog, two on pegvisomant, and two on pegvisomant and a somatostatin analog. After six months of dose escalation or the addition of pegvisomant, IGF1 decreased from 1.22 ± 0.14 to 0.87 ± 0.20 times the upper limit of normal (p=0.001). PASQ score decreased by 3.5 (p=0.02) and the ACRODAT® overall status decreased by 50.5 (p=0.001); however, there was no difference in the AcroQoL score. Hemoglobin A1c and liver enzymes did not differ and repeat MRI of the sella at 6 months showed no change.ConclusionsIn this pilot study, stricter control of modest IGF1 elevations led to symptomatic improvement as measured by the PASQ score. These findings prompt larger prospective trials. |
| format | Article |
| id | doaj-art-a0334af108ee46fabad5e8c1fdfde232 |
| institution | Kabale University |
| issn | 1664-2392 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | Frontiers Media S.A. |
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| series | Frontiers in Endocrinology |
| spelling | doaj-art-a0334af108ee46fabad5e8c1fdfde2322025-01-31T05:10:24ZengFrontiers Media S.A.Frontiers in Endocrinology1664-23922025-01-011610.3389/fendo.2025.15168991516899Impact of strict IGF1 control on quality-of-life scores in patients with acromegalyChaitanya Gandhi0Marie-Claire Denis1Daniel Holmes2Juan Rivera3Stan van Uum4Shereen Ezzat5Constance Chik6Department of Medicine, University of Alberta, Edmonton, AB, CanadaDepartment of Medicine, Centre Hospitalier de l’Université Laval, Quebec City, QC, CanadaDepartment of Pathology and Laboratory Medicine, St Paul’s Hospital, Vancouver, BC, CanadaDepartment of Medicine, McGill University, Montreal, QC, CanadaDepartment of Medicine, Western University, London, ON, CanadaDepartment of Medicine, Princess Margaret Cancer Centre, Toronto, ON, CanadaDepartment of Medicine, University of Alberta, Edmonton, AB, CanadaObjectiveExamine, in a real-world setting, whether strict normalization of modestly elevated insulin-like growth factor 1 (IGF1) results in clinical and health-related quality of life benefits in patients with acromegaly using an open-label, non-randomized, 6-month prospective interventional study.MethodsIn patients with acromegaly and modest IGF1 elevation, strict IGF1 control was achieved by addition or dose escalation of pegvisomant. Clinical and biochemical parameters were assessed at baseline, 1 and 3 months for pegvisomant dose titration, and at 6 months. The Patient-Assessed Acromegaly Symptom Questionnaire (PASQ), the Acromegaly Quality of Life questionnaire (AcroQoL) and the Acromegaly Disease Activity Tool (ACRODAT®) were completed at baseline and at 6 months.ResultsTen patients (8 males) with mean age of 50.7 years participated in the study. All patients had a macroadenoma and nine had prior transsphenoidal surgeries. At time of screening, six patients were on a somatostatin analog, two on pegvisomant, and two on pegvisomant and a somatostatin analog. After six months of dose escalation or the addition of pegvisomant, IGF1 decreased from 1.22 ± 0.14 to 0.87 ± 0.20 times the upper limit of normal (p=0.001). PASQ score decreased by 3.5 (p=0.02) and the ACRODAT® overall status decreased by 50.5 (p=0.001); however, there was no difference in the AcroQoL score. Hemoglobin A1c and liver enzymes did not differ and repeat MRI of the sella at 6 months showed no change.ConclusionsIn this pilot study, stricter control of modest IGF1 elevations led to symptomatic improvement as measured by the PASQ score. These findings prompt larger prospective trials.https://www.frontiersin.org/articles/10.3389/fendo.2025.1516899/fullacromegalyquality-of-lifepegvisomantinsulin-like growth factor 1clinical trial |
| spellingShingle | Chaitanya Gandhi Marie-Claire Denis Daniel Holmes Juan Rivera Stan van Uum Shereen Ezzat Constance Chik Impact of strict IGF1 control on quality-of-life scores in patients with acromegaly Frontiers in Endocrinology acromegaly quality-of-life pegvisomant insulin-like growth factor 1 clinical trial |
| title | Impact of strict IGF1 control on quality-of-life scores in patients with acromegaly |
| title_full | Impact of strict IGF1 control on quality-of-life scores in patients with acromegaly |
| title_fullStr | Impact of strict IGF1 control on quality-of-life scores in patients with acromegaly |
| title_full_unstemmed | Impact of strict IGF1 control on quality-of-life scores in patients with acromegaly |
| title_short | Impact of strict IGF1 control on quality-of-life scores in patients with acromegaly |
| title_sort | impact of strict igf1 control on quality of life scores in patients with acromegaly |
| topic | acromegaly quality-of-life pegvisomant insulin-like growth factor 1 clinical trial |
| url | https://www.frontiersin.org/articles/10.3389/fendo.2025.1516899/full |
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