Skip to main content
Hauptrubriken
CME Facharzt-Training Webinare Zeitschriften Bücher e.Medpedia Podcast
Service
Jobbörse Info & Hilfe
Fachgebiete
Anästhesiologie Allgemeinmedizin Arbeitsmedizin Augenheilkunde Chirurgie Dermatologie Gynäkologie und Geburtshilfe HNO Innere Medizin Kardiologie Neurologie Onkologie und Hämatologie Orthopädie und Unfallchirurgie Pädiatrie Pathologie Psychiatrie Radiologie Rechtsmedizin Urologie Zahnmedizin
Themen
Klimawandel und Gesundheit Neues aus dem Markt COVID-19 Praxis und Beruf Seltene Erkrankungen GOÄ & EBM Panorama
Sammlungen
Kasuistiken Algorithmen & Infografiken Blickdiagnosen Arthropedia FlapFinder (für Dermatochirurgen) Videos Kongressberichterstattung Medizin für Apothekerinnen und Apotheker Für Ärztinnen und Ärzte in Weiterbildung Für Medizinstudierende
Erweiterte Suche
Anmelden
nach oben
Current Treatment Options in Oncology
Erschienen in: Current Treatment Options in Oncology 4/2014

01.12.2014 | Neuro-oncology (GJ Lesser, Section Editor)

Patient Reported Endpoints for Measuring Clinical Benefit in (High Grade Glioma) Primary Brain Tumor Patients

verfasst von: Terri S. Armstrong, PhD, Mark R. Gilbert, MD

Erschienen in: Current Treatment Options in Oncology | Ausgabe 4/2014

Einloggen, um Zugang zu erhalten

Opinion statement

Symptom occurrence impacts primary brain tumor patients from the time of diagnosis and often heralds recurrence. In addition, the therapy can also result in symptoms that may compound tumor-associated symptoms, further impacting the patient’s function and overall quality of life. There is increasing recognition that clinical studies evaluating tumor response using only measures of tumor size on imaging or survival are inadequate in brain tumor patients. Many symptoms can only be assessed from the patient, and patient reported outcome measures have been developed and have adequate reliability and validity. These measures are beginning to be incorporated into clinical trials. Guidelines on their use and meaning are needed to standardize assessment across trials and facilitate measurement of clinical benefit.
Literatur
1.
Johnson JR, Ning YM, Farrell A, Justice R, Keegan P, Pazdur R. Accelerated approval of oncology products: the food and drug administration experience. J Natl Cancer Inst. 2011;103(8):636–44.PubMedCrossRef
2.
Wildiers H, Mauer M, Pallis A, et al. End points and trial design in geriatric oncology research: a joint European organisation for research and treatment of cancer–Alliance for Clinical Trials in Oncology–International Society Of Geriatric Oncology position article. J Clin Oncol. 2013;31(29):3711–8.PubMedCrossRef
3.
Pazdur R. Endpoints for assessing drug activity in clinical trials. Oncologist. 2008;13 Suppl 2:19–21.PubMedCrossRef
4.
Ostrom QT, Gittleman H, Farah P, et al. CBTRUS statistical report: primary brain and central nervous system tumors diagnosed in the United States in 2006–2010. Neuro Oncol. 2013;15 Suppl 2:ii1–56.PubMedCrossRef
5.
Stupp R, Hegi ME, Mason WP, et al. Effects of radiotherapy with concomitant and adjuvant temozolomide versus radiotherapy alone on survival in glioblastoma in a randomised phase III study: 5-year analysis of the EORTC-NCIC trial. Lancet Oncol. 2009;10(5):459–66.PubMedCrossRef
6.
Sherwood PR, Donovan HS, Given CW, et al. Predictors of employment and lost hours from work in cancer caregivers. Psychooncology. 2008;17(6):598–605.PubMedCrossRef
7.
Armstrong TS, Vera-Bolanos E, Gilbert MR. Clinical course of adult patients with ependymoma: results of the Adult Ependymoma Outcomes Project. Cancer. 2011;117(22):5133–41.PubMedCrossRef
8.
Salander P, Bergenheim AT, Henriksson R. How was life after treatment of a malignant brain tumour? Soc Sci Med. 2000;51(4):589–98.PubMedCrossRef
9.
Bradley S, Sherwood PR, Donovan HS, et al. I could lose everything: understanding the cost of a brain tumor. J Neurooncol. 2007;85(3):329–38.PubMedCrossRef
10.
Yabroff KR, Harlan L, Zeruto C, Abrams J, Mann B. Patterns of care and survival for patients with glioblastoma multiforme diagnosed during 2006. Neuro Oncol. 2012;14(3):351–9.PubMedCrossRefPubMedCentral
11.
Stupp R, Mason WP, van den Bent MJ, et al. Radiotherapy plus concomitant and adjuvant temozolomide for glioblastoma. N Engl J Med. 2005;352(10):987–96.PubMedCrossRef
12.
Brandes AA, Tosoni A, Spagnolli F, et al. Disease progression or pseudoprogression after concomitant radiochemotherapy treatment: pitfalls in neurooncology. Neuro Oncol. 2008;10(3):361–7.PubMedCrossRefPubMedCentral
13.
Fink J, Born D, Chamberlain MC. Pseudoprogression: relevance with respect to treatment of high-grade gliomas. Curr Treat Options Oncol. 2011;12(3):240–52.PubMedCrossRef
14.
Chamberlain MC. Pseudoprogression in glioblastoma. J Clin Oncol. 2008;26(26):4359. author reply –60.
15.
Arpinelli F, Bamfi F. The FDA guidance for industry on PROs: the point of view of a pharmaceutical company. Health Qual Life Outcomes. 2006;4:85.PubMedCrossRefPubMedCentral
16.
Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance. Health Qual Life Outcomes 2006;4:79.
17.
Reilly CM, Bruner DW, Mitchell SA, et al. A literature synthesis of symptom prevalence and severity in persons receiving active cancer treatment. Support Care Cancer. 2013;21(6):1525–50.PubMedCrossRef
18.
Cleeland CS, Zhao F, Chang VT, et al. The symptom burden of cancer: evidence for a core set of cancer-related and treatment-related symptoms from the Eastern Cooperative Oncology Group Symptom Outcomes and Practice Patterns study. Cancer. 2013;119(24):4333–40.PubMedCrossRef
19.
Verstovsek S, Mesa RA, Gotlib J, et al. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med. 2012;366(9):799–807.PubMedCrossRef
20.
Mesa RA, Gotlib J, Gupta V, et al. Effect of ruxolitinib therapy on myelofibrosis-related symptoms and other patient-reported outcomes in COMFORT-I: a randomized, double-blind, placebo-controlled trial. J Clin Oncol. 2013;31(10):1285–92.PubMedCrossRef
21.
Mauer ME, Bottomley A, Taphoorn MJ. Evaluating health-related quality of life and symptom burden in brain tumour patients: instruments for use in experimental trials and clinical practice. Curr Opin Neurol. 2008;21(6):745–53.PubMedCrossRef
22.
Henriksson R, Asklund T, Poulsen HS. Impact of therapy on quality of life, neurocognitive function and their correlates in glioblastoma multiforme: a review. J Neurooncol. 2011;104(3):639–46.PubMedCrossRefPubMedCentral
23.
Mauer M, Stupp R, Taphoorn MJ, et al. The prognostic value of health-related quality-of-life data in predicting survival in glioblastoma cancer patients: results from an international randomised phase III EORTC Brain Tumour and Radiation Oncology Groups, and NCIC Clinical Trials Group study. Br J Cancer. 2007;97(3):302–7.PubMedCrossRefPubMedCentral
24.•
Armstrong TS, Wefel JS, Wang M, et al. Net clinical benefit analysis of radiation therapy oncology group 0525: a phase III trial comparing conventional adjuvant temozolomide with dose-intensive temozolomide in patients with newly diagnosed glioblastoma. J Clin Oncol. 2013;31(32):4076–84. Large clinical study first reporting the use of ‘Net Clinical Benefit’ endpoints.PubMedCrossRef
25.••
Gilbert MR, Dignam JJ, Armstrong TS, et al. A randomized trial of bevacizumab for newly diagnosed glioblastoma. N Engl J Med. 2014;370(8):699–708. Randomized placebo controlled trial using patient-reported outcomes and objective testing showing worsening of symptoms when imaging delcared progression free.PubMedCrossRefPubMedCentral
26.••
Chinot OL, Wick W, Cloughesy T. Bevacizumab for newly diagnosed glioblastoma. N Engl J Med. 2014;370(21):2049. Randomized placebo controlled trial using HRQOL patient reported outcomes showing longer maintenance of HRQOL with bevacizumab.PubMedCrossRef
27.
Armstrong TS, Mendoza T, Gning I, et al. Validation of the M.D. Anderson Symptom Inventory Brain Tumor Module (MDASI-BT). J Neurooncol. 2006;80(1):27–35.PubMedCrossRef
28.
Armstrong TS, Vera-Bolanos E, Gning I, et al. The impact of symptom interference using the MD Anderson Symptom Inventory-Brain Tumor Module (MDASI-BT) on prediction of recurrence in primary brain tumor patients. Cancer. 2011;117(14):3222–8.PubMedCrossRef
29.
Dirven L, Reijneveld JC, Aaronson NK, Bottomley A, Uitdehaag BM, Taphoorn MJ. Health-related quality of life in patients with brain tumors: limitations and additional outcome measures. Curr Neurol Neurosci Rep. 2013;13(7):359.PubMedCrossRef
30.
Sikkes SA, de Lange-de Klerk ES, Pijnenburg YA, et al. A new informant-based questionnaire for instrumental activities of daily living in dementia. Alzheimers Dement. 2012;8(6):536–43.PubMedCrossRef
31.
Adamis D. Statistical methods for analysing longitudinal data in delirium studies. Int Rev Psychiatry. 2009;21(1):74–85.PubMedCrossRef
32.
Armstrong TS. 2013 Special Topics Conference: peaks and pitfalls in longitudinal studies of symptom outcome data. Nursing Outlook [In press].
33.
Armstrong TS. Measuring clinical benefit: use of patient-reported outcomes (PRO) in primary brain tumor clinical trials. Curr Oncol Rep. 2013;15(1):27–32.PubMedCrossRef
34.
FDA. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance. Health Qual Life Outcomes. 2006;4:79.CrossRef
35.
36.
Lavori PW, Brown CH, Duan N, Gibbons RD, Greenhouse J. Missing data in longitudinal clinical trials part A: design and conceptual issues. Psychiatr Ann. 2008;38(12):784–92.PubMedCrossRefPubMedCentral
37.
Saunders JA, Morrow-Howell N, Spitznagel E, Dore P, Proctor EK, Pescarino R. Imputing missing data: a comparison of methods for social work researchers. Soc Work Res. 2006;30(1):19–31.CrossRef
38.
Tabachnick BG, Fidell LS. Cleaning up your act: screening data prior to analysis. In: Tabachnick BG, Fidell LS, editors. Using multivariate statistics. New York: Harper and Row; 1983.
39.
Curran D, Molenberghs G, Fayers PM, Machin D. Incomplete quality of life data in randomized trials: missing forms. Stat Med. 1998;17(5–7):697–709.PubMedCrossRef
40.
Armstrong TS, Vera-Bolanos E, Acquaye A, Gilbert MR, Mendoza TR. Impact of recall period on primary brain tumor patient's self report of symptoms. Neurooncol Pract. 2014.
41.
Brown PD, Decker PA, Rummans TA, et al. A prospective study of quality of life in adults with newly diagnosed high-grade gliomas: comparison of patient and caregiver ratings of quality of life. Am J Clin Oncol. 2008;31(2):163–8.PubMedCrossRef
42.
Burris H, Storniolo AM. Assessing clinical benefit in the treatment of pancreas cancer: gemcitabine compared to 5-fluorouracil. Eur J Cancer. 1997;33 Suppl 1:S18–22.PubMedCrossRef
43.
Burris 3rd HA, Moore MJ, Andersen J, et al. Improvements in survival and clinical benefit with gemcitabine as first-line therapy for patients with advanced pancreas cancer: a randomized trial. J Clin Oncol. 1997;15(6):2403–13.PubMed
44.
Esther Kim JE, Dodd MJ, Aouizerat BE, Jahan T, Miaskowski C. A review of the prevalence and impact of multiple symptoms in oncology patients. J Pain Symptom Manag. 2009;37(4):715–36.CrossRef
45.
Basch E, Abernethy AP, Mullins CD, et al. Recommendations for incorporating patient-reported outcomes into clinical comparative effectiveness research in adult oncology. J Clin Oncol. 2012;30(34):4249–55.PubMedCrossRef
46.
Basch E. Beyond the FDA PRO guidance: steps toward integrating meaningful patient-reported outcomes into regulatory trials and US drug labels. Value Health. 2012;15(3):401–3.PubMedCrossRef
47.
Bruner DW, Movsas B, Basch E. Capturing the patient perspective: patient-reported outcomes as clinical trial endpoints. Am Soc Clin Oncol Educ Book. 2012;32:139–44.
Metadaten
Titel
Patient Reported Endpoints for Measuring Clinical Benefit in (High Grade Glioma) Primary Brain Tumor Patients
verfasst von
Terri S. Armstrong, PhD
Mark R. Gilbert, MD
Publikationsdatum
01.12.2014
Verlag
Springer US
Erschienen in
Current Treatment Options in Oncology / Ausgabe 4/2014
Print ISSN: 1527-2729
Elektronische ISSN: 1534-6277
DOI
https://doi.org/10.1007/s11864-014-0302-8

Weitere Artikel der Ausgabe 4/2014

Current Treatment Options in Oncology 4/2014 Zur Ausgabe

Lung Cancer (HA Wakelee, Section Editor)

The Minority Report: Targeting the Rare Oncogenes in NSCLC

Neuro-oncology (GJ Lesser, Section Editor)

Current Role of Anti-Angiogenic Strategies for Glioblastoma

Neuro-oncology (GJ Lesser, Section Editor)

Treatment of Radiation-Induced Cognitive Decline

Head and Neck Cancer (J-P Machiels, Section Editor)

Interventional Pulmonologist Perspective: Treatment of Malignant Pleural Effusion

Head and Neck Cancer (J-P Machiels, Section Editor)

Human Papillomavirus-Associated Oropharynx Cancer (HPV-OPC): Treatment Options

Neuro-oncology (GJ Lesser, Section Editor)

Therapeutic Strategies for Cancer Treatment Related Peripheral Neuropathies

Neu im Fachgebiet Onkologie

Adjuvante Immuntherapie verlängert Leben bei RCC

25.04.2024 Nierenkarzinom Nachrichten

Nun gibt es auch Resultate zum Gesamtüberleben: Eine adjuvante Pembrolizumab-Therapie konnte in einer Phase-3-Studie das Leben von Menschen mit Nierenzellkarzinom deutlich verlängern. Die Sterberate war im Vergleich zu Placebo um 38% geringer.

Alectinib verbessert krankheitsfreies Überleben bei ALK-positivem NSCLC

25.04.2024 NSCLC Nachrichten

Das Risiko für Rezidiv oder Tod von Patienten und Patientinnen mit reseziertem ALK-positivem NSCLC ist unter einer adjuvanten Therapie mit dem Tyrosinkinase-Inhibitor Alectinib signifikant geringer als unter platinbasierter Chemotherapie.

Bei Senioren mit Prostatakarzinom auf Anämie achten!

24.04.2024 DGIM 2024 Nachrichten

Patienten, die zur Behandlung ihres Prostatakarzinoms eine Androgendeprivationstherapie erhalten, entwickeln nicht selten eine Anämie. Wer ältere Patienten internistisch mitbetreut, sollte auf diese Nebenwirkung achten.

ICI-Therapie in der Schwangerschaft wird gut toleriert

23.04.2024 Medikamente in Schwangerschaft und Stillzeit Nachrichten

Müssen sich Schwangere einer Krebstherapie unterziehen, rufen Immuncheckpointinhibitoren offenbar nicht mehr unerwünschte Wirkungen hervor als andere Mittel gegen Krebs.

Update Onkologie

Bestellen Sie unseren Fach-Newsletter und bleiben Sie gut informiert.

Newsletter bestellen
Bildnachweise