nach oben

Current Hematologic Malignancy Reports

Erschienen in:

14.01.2019 | Stem Cell Transplantation (R Maziarz, Section Editor)

Graft Versus Host Disease Clinical Trials: Is it Time for Patients Centered Outcomes to Be the Primary Objective?

verfasst von: Bronwen E. Shaw

Erschienen in: Current Hematologic Malignancy Reports | Ausgabe 1/2019

Einloggen, um Zugang zu erhalten

Abstract

Purpose of Review

Graft versus host disease (GVHD) is a common complication following hematopoietic cell transplant and is associated with a high symptom burden, reduced functional status, and impaired quality of life (QOL). QOL is best assessed by patient-reported outcomes (PRO). Numerous clinical trials for the prevention and treatment of GVHD are available. This review aims to understand the landscape of PRO inclusion in clinical trials for GVHD over the last decade.

Recent Findings

Consensus bodies, including experts in GVHD, PRO, and clinical trials have made recommendations for a standardized approach for the inclusion of PRO in clinical trials including as primary outcomes, however, these have yet to be implemented in a consistent manner in practice.

Summary

Consistently applying consensus recommendation in chronic GVHD will ensure that PROs are appropriately included in clinical trials. Development of validated measures in acute GVHD and composite outcomes for all GVHD trials are required.

Brundage M, Bass B, Davidson J, Queenan J, Bezjak A, Ringash J, et al. Patterns of reporting health-related quality of life outcomes in randomized clinical trials: implications for clinicians and quality of life researchers. Qual Life Res. 2011;20(5):653–64.CrossRefPubMed

Lee SJ, Williams LA. Patient-reported outcomes for acute graft-versus-host disease prevention and treatment trials. Biol Blood Marrow Transplant. 2010;16(3):295–300.CrossRefPubMed

Martin PJ, Weisdorf D, Przepiorka D, Hirschfeld S, Farrell A, Rizzo JD, et al. National Institutes of Health Consensus Development Project On Criteria For Clinical Trials in chronic graft-versus-host disease: VI. Design of Clinical Trials Working Group report. Biol Blood Marrow Transplant. 2006;12(5):491–505.CrossRefPubMed

FDA. US Food and Drug Administration. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims. US Department of Health and Human Services Food and Drug Administration. 2009. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM193282.pdf. Accessed 30 Oct 2018.

Rothrock NE, Kaiser KA, Cella D. Developing a valid patient-reported outcome measure. Clin Pharmacol Ther. 2011;90(5):737–42.CrossRefPubMed

McQuellon RP, Russell GB, Cella DF, Craven BL, Brady M, Bonomi A, et al. Quality of life measurement in bone marrow transplantation: development of the functional assessment of cancer therapy-bone marrow transplant (FACT-BMT) scale. Bone Marrow Transplant. 1997;19(4):357–68.CrossRefPubMed

Cleeland CS, Mendoza TR, Wang XS, Chou C, Harle MT, Morrissey M, et al. Assessing symptom distress in cancer patients: the M.D. Anderson symptom inventory. Cancer. 2000;89(7):1634–46.CrossRefPubMed

Ware JE Jr, Sherbourne CD. The MOS 36-item short-form health survey (SF-36). I. Conceptual framework and item selection. Med Care. 1992;30(6):473–83.CrossRefPubMed

Herdman M, Gudex C, Lloyd A, Janssen M, Kind P, Parkin D, et al. Development and preliminary testing of the new five-level version of EQ-5D (EQ-5D-5L). Qual Life Res. 2011;20(10):1727–36.CrossRefPubMedCentralPubMed

10.

Lee S, Cook EF, Soiffer R, Antin JH. Development and validation of a scale to measure symptoms of chronic graft-versus-host disease. Biol Blood Marrow Transplant. 2002;8(8):444–52.CrossRefPubMed

11.

Daughton DM, Fix AJ, Kass I, Bell CW, Patil KD. Maximum oxygen consumption and the ADAPT quality-of-life scale. Arch Phys Med Rehabil. 1982;63(12):620–2.PubMed

12.

Hoodin F, LaLonde L, Errickson J, Votruba K, Kentor R, Gatza E, et al. Cognitive function and quality of life in vorinostat-treated patients after matched unrelated donor myeloablative conditioning hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2018. https://doi.org/10.1016/j.bbmt.2018.09.015.

13.

Bolanos-Meade J, Logan BR, Alousi AM, Antin JH, Barowski K, Carter SL, et al. Phase 3 clinical trial of steroids/mycophenolate mofetil vs steroids/placebo as therapy for acute GVHD: BMT CTN 0802. Blood. 2014;124(22):3221–7 quiz 335.CrossRefPubMedCentralPubMed

14.

Carpenter PA, Logan BR, Lee SJ, Weisdorf DJ, Johnston L, Costa LJ, et al. A phase II/III randomized, multicenter trial of prednisone /sirolimus vs prednisone/sirolimus/calcineurin inhibitor for treatment of chronic graft-versus-host disease: BMT CTN 0801. Haematologica. 2018;103:1915–24.CrossRefPubMedCentralPubMed

15.

Baird K, Comis LE, Joe GO, Steinberg SM, Hakim FT, Rose JJ, et al. Imatinib mesylate for the treatment of steroid-refractory sclerotic-type cutaneous chronic graft-versus-host disease. Biol Blood Marrow Transplant. 2015;21(6):1083–90.CrossRefPubMedCentralPubMed

16.

Cutler C, Miklos D, Kim HT, Treister N, Woo SB, Bienfang D, et al. Rituximab for steroid-refractory chronic graft-versus-host disease. Blood. 2006;108(2):756–62.CrossRefPubMedCentralPubMed

17.

St John L, Gordon SM, Childs R, Marquesen M, Pavletic SZ, Wu TX, et al. Topical thalidomide gel in oral chronic GVHD and role of in situ cytokine expression in monitoring biological activity. Bone Marrow Transplant. 2013;48(4):610–1.CrossRefPubMed

18.

DeFilipp Z, Nazarian RM, El-Jawahri A, Li S, Brown J, Del Rio C, et al. Phase 1 study of the hedgehog pathway inhibitor sonidegib for steroid-refractory chronic graft-versus-host disease. Blood Adv. 2017;1(22):1919–22.CrossRefPubMedCentralPubMed

19.

Pavletic SZ. Response as an end point in treatment trials for acute GVHD. Bone Marrow Transplant. 2012;47(2):161–3.CrossRefPubMed

20.

Harris AC, Young R, Devine S, Hogan WJ, Ayuk F, Bunworasate U, et al. International, multicenter standardization of acute graft-versus-host disease clinical data collection: a report from the Mount Sinai Acute gvhd International Consortium. Biol Blood Marrow Transplant. 2016;22(1):4–10.CrossRefPubMed

21.

Schoemans HM, Lee SJ, Ferrara JL, Wolff D, Levine JE, Schultz KR, et al. EBMT-NIH-CIBMTR Task Force position statement on standardized terminology & guidance for graft-versus-host disease assessment. Bone Marrow Transplant. 2018;53(11):1401–15.CrossRefPubMedPubMedCentral

22.

Chronic GC. Rationale and design of the chronic GVHD cohort study: improving outcomes assessment in chronic GVHD. Biol Blood Marrow Transplant. 2011;17(8):1114–20.CrossRef

23.

•• Pidala J, Kurland B, Chai X, Majhail N, Weisdorf DJ, Pavletic S, et al. Patient-reported quality of life is associated with severity of chronic graft-versus-host disease as measured by NIH criteria: report on baseline data from the Chronic GVHD Consortium. Blood. 2011;117(17):4651–7 This paper shows that cGVHD severity is independently associated with Quality of Life. CrossRefPubMedCentralPubMed

24.

Pidala J, Kurland BF, Chai X, Vogelsang G, Weisdorf DJ, Pavletic S, et al. Sensitivity of changes in chronic graft-versus-host disease activity to changes in patient-reported quality of life: results from the chronic graft-versus-host disease consortium. Haematologica. 2011;96(10):1528–35.CrossRefPubMedCentralPubMed

25.

Inamoto Y, Chai X, Kurland BF, Cutler C, Flowers ME, Palmer JM, et al. Validation of measurement scales in ocular graft-versus-host disease. Ophthalmology. 2012;119(3):487–93.CrossRefPubMed

26.

Inamoto Y, Martin PJ, Chai X, Jagasia M, Palmer J, Pidala J, et al. Clinical benefit of response in chronic graft-versus-host disease. Biol Blood Marrow Transplant. 2012;18(10):1517–24.CrossRefPubMedCentralPubMed

27.

Jacobsohn DA, Rademaker A, Kaup M, Vogelsang GB. Skin response using NIH consensus criteria vs Hopkins scale in a phase II study for steroid-refractory chronic GVHD. Bone Marrow Transplant. 2009;44(12):813–9.CrossRefPubMed

28.

Palmer J, Williams K, Inamoto Y, Chai X, Martin PJ, Tomas LS, et al. Pulmonary symptoms measured by the national institutes of health lung score predict overall survival, nonrelapse mortality, and patient-reported outcomes in chronic graft-versus-host disease. Biol Blood Marrow Transplant. 2014;20(3):337–44.CrossRefPubMed

29.

Treister N, Chai X, Kurland B, Pavletic S, Weisdorf D, Pidala J, et al. Measurement of oral chronic GVHD: results from the Chronic GVHD consortium. Bone Marrow Transplant. 2013;48(8):1123–8.CrossRefPubMedCentralPubMed

30.

Merkel EC, Mitchell SA, Lee SJ. Content validity of the Lee chronic graft-versus-host disease symptom scale as assessed by cognitive interviews. Biol Blood Marrow Transplant. 2016;22(4):752–8.CrossRefPubMedCentralPubMed

31.

Martin PJ, Lee SJ, Przepiorka D, Horowitz MM, Koreth J, Vogelsang GB, et al. National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: VI. The 2014 clinical trial design working group report. Biol Blood Marrow Transplant. 2015;21(8):1343–59.CrossRefPubMedCentralPubMed

32.

•• Lee SJ, Wolff D, Kitko C, Koreth J, Inamoto Y, Jagasia M, et al. Measuring therapeutic response in chronic graft-versus-host disease. National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: IV. The 2014 response criteria working group report. Biol Blood Marrow Transplant. 2015;21(6):984–99 This consensus document describes the use of the LEE symptom scale as a chronic-GVHD core measure to use in cGVHD clinical trials. CrossRefPubMedCentralPubMed

33.

Pidala J, Chai X, Kurland BF, Inamoto Y, Flowers ME, Palmer J, et al. Analysis of gastrointestinal and hepatic chronic graft-versus-host [corrected] disease manifestations on major outcomes: a chronic graft-versus-host [corrected] disease consortium study. Biol Blood Marrow Transplant. 2013;19(5):784–91.CrossRefPubMedCentralPubMed

34.

Palmer J, Chai X, Pidala J, Inamoto Y, Martin PJ, Storer B, et al. Predictors of survival, nonrelapse mortality, and failure-free survival in patients treated for chronic graft-versus-host disease. Blood. 2016;127(1):160–6.CrossRefPubMedCentralPubMed

35.

Efficace F, Gaidano G, Lo-Coco F. Patient-reported outcomes in hematology: is it time to focus more on them in clinical trials and hematology practice? Blood. 2017;130(7):859–66.CrossRefPubMed

36.

Efficace F, Fayers P, Pusic A, Cemal Y, Yanagawa J, Jacobs M, et al. Quality of patient-reported outcome reporting across cancer randomized controlled trials according to the CONSORT patient-reported outcome extension: a pooled analysis of 557 trials. Cancer. 2015;121(18):3335–42.CrossRefPubMed

37.

Chan AW, Tetzlaff JM, Altman DG, Laupacis A, Gotzsche PC, Krleza-Jeric K, et al. SPIRIT 2013 statement: defining standard protocol items for clinical trials. Ann Intern Med. 2013;158(3):200–7.CrossRefPubMedCentralPubMed

38.

Chan AW, Tetzlaff JM, Gotzsche PC, Altman DG, Mann H, Berlin JA, et al. SPIRIT 2013 explanation and elaboration: guidance for protocols of clinical trials. BMJ. 2013;346:e7586.CrossRefPubMedCentralPubMed

39.

Calvert M, Kyte D, Mercieca-Bebber R, Slade A, Chan AW, King MT, et al. Guidelines for inclusion of patient-reported outcomes in clinical trial protocols: the SPIRIT-PRO extension. JAMA. 2018;319(5):483–94.CrossRefPubMed

40.

Moher D, Hopewell S, Schulz KF, Montori V, Gotzsche PC, Devereaux PJ, et al. CONSORT 2010 explanation and elaboration: updated guidelines for reporting parallel group randomised trials. BMJ. 2010;340:c869.CrossRefPubMedCentralPubMed

41.

Calvert M, Blazeby J, Altman DG, Revicki DA, Moher D, Brundage MD, et al. Reporting of patient-reported outcomes in randomized trials: the CONSORT PRO extension. JAMA. 2013;309(8):814–22.CrossRefPubMed

42.

CONSORT. http://www.consort-statement.org/extensions?ContentWidgetId=560, Accessed 30 Oct 2018.

43.

Appelbaum FR, Anasetti C, Antin JH, Atkins H, Davies S, Devine S, et al. Blood and marrow transplant clinical trials network state of the science symposium 2014. Biol Blood Marrow Transplant. 2015;21(2):202–24.CrossRefPubMed

44.

Shaw BE, Lee SJ, Horowitz MM, Wood WA, Rizzo JD, Flynn KE. Can we agree on patient-reported outcome measures for assessing hematopoietic cell transplantation patients? A study from the CIBMTR and BMT CTN. Bone Marrow Transplant. 2016;51(9):1173–9.CrossRefPubMed

45.

Shaw BE, Syrjala KL, Onstad LE, Chow EJ, Flowers ME, Jim H, et al. PROMIS measures can be used to assess symptoms and function in long-term hematopoietic cell transplantation survivors. Cancer. 2018;124(4):841–9.CrossRefPubMed

46.

Lee SJ, Onstad L, Chow EJ, Shaw BE, Jim HSL, Syrjala KL, et al. Patient-reported outcomes and health status associated with chronic graft-versus-host disease. Haematologica. 2018;103(9):1535–41.CrossRefPubMedCentralPubMed

47.

PROCTCAE. https://healthcaredelivery.cancer.gov/pro-ctcae/. Accessed 30 Oct 2018.

48.

Wood WA, Deal AM, Abernethy A, Basch E, Battaglini C, Kim YH, et al. Feasibility of frequent patient-reported outcome surveillance in patients undergoing hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2013;19(3):450–9.CrossRefPubMed

Titel: Graft Versus Host Disease Clinical Trials: Is it Time for Patients Centered Outcomes to Be the Primary Objective?
verfasst von: Bronwen E. Shaw
Publikationsdatum: 14.01.2019
Verlag: Springer US
Erschienen in: Current Hematologic Malignancy Reports / Ausgabe 1/2019
Print ISSN: 1558-8211
Elektronische ISSN: 1558-822X
DOI: https://doi.org/10.1007/s11899-019-0494-x

Neu im Fachgebiet Onkologie

24.04.2024 | DGIM 2024 | Nachrichten

Springer Medizin

Graft Versus Host Disease Clinical Trials: Is it Time for Patients Centered Outcomes to Be the Primary Objective?

Abstract

Purpose of Review

Recent Findings

Summary

Neu im Fachgebiet Onkologie

Bei Senioren mit Prostatakarzinom auf Anämie achten!

ICI-Therapie in der Schwangerschaft wird gut toleriert

Krebspatienten impfen: Was? Wen? Und wann nicht?

Müde im Alter? Auch an die Nebenniere denken

Update Onkologie

Springer Medizin

Abstract

Purpose of Review

Recent Findings

Summary

Bitte loggen Sie sich ein, um Zugang zu diesem Inhalt zu erhalten

Weitere Artikel der Ausgabe 1/2019

Should Overall Survival Remain an Endpoint for Multiple Myeloma Trials?

Industry’s Giant Leap Into Cellular Therapy: Catalyzing Chimeric Antigen Receptor T Cell (CAR-T) Immunotherapy

Treatment-Free Remission in CML: the US Perspective

Immunotherapeutic Approaches for Multiple Myeloma: Where Are We Now?

Determination of Minimal Residual Disease in Multiple Myeloma: Does It Matter?

The Emerging Role of Liquid Biopsies in Lymphoproliferative Disorders

Neu im Fachgebiet Onkologie

Bei Senioren mit Prostatakarzinom auf Anämie achten!

ICI-Therapie in der Schwangerschaft wird gut toleriert

Krebspatienten impfen: Was? Wen? Und wann nicht?

Müde im Alter? Auch an die Nebenniere denken

Update Onkologie