Defining Clinically Meaningful Outcomes in the Evaluation of New Treatments for Oral Mucositis: Oral Mucositis Patient Provider Advisory Board

Bellm, Lisa A.; Cunningham, Gail; Durnell, Laurie; Eilers, June; Epstein, Joel B.; Fleming, Terence J.; Fuchs, Henry J.; Haskins, Martha Nash; Horowitz, Mary M.; Martin, Paul J.; McGuire, Deborah B.; Mullane, Kevin; Oster, Gerry

doi:10.1081/cnv-120002497

Cited by 59 publications

(28 citation statements)

References 31 publications

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“…These results strongly suggest that the MTS components of the OMDQ may enable earlier detection of OM and may lead to changes in therapy, including earlier initiation of analgesics. 7 Further evaluation of this OMDQ in other clinical settings involving OM is ongoing.…”

Section: Discussionmentioning

confidence: 99%

“…7 Early versions of the OMDQ were administered in phase 1 clinical trials (both in hematological and solid tumor settings) receiving high-dose BEAM and 5-FU/Leucovorin treatment regimens. Results from these studies showed that the OMDQ correlated positively with clinically measured assessments of OM (r ¼ 0.2-0.5 for the MTS vs RTOG comparisons; unpublished data).…”

Section: Oral Mucositis Daily Questionnairementioning

confidence: 99%

“…It has been recommended that clinical trials of new interventions for OM should include objective assessment of patient-reported outcomes. 7 As shown in Table 1, there are several established instruments for the clinical assessment of OM severity, including scales from the World Health Organization (WHO), the Radiation Therapy Oncology Group (RTOG), and the Western Consortium for Cancer Nursing Research (WCCNR), among others. [8][9][10][11][12][13] Currently, there is no established patient reported questionnaire with demonstrated reliability and validity for patient self-assessment of the signs and symptoms of OM.…”

Section: Introductionmentioning

confidence: 99%

“…It was developed through a series of focus groups and one-on-one interviews with cancer patients and was later refined after preliminary versions were used in phase 1 and 2 trials of palifermin. 7 An objective of this study was to evaluate and confirm the psychometric properties (feasibility, reliability, validity, and responsiveness) of the patient-reported OMDQ. We also compared the OMDQ results with those results from established clinical scales rated by clinicians in this pivotal phase 3 clinical trial of palifermin.…”

Section: Introductionmentioning

confidence: 99%

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Reliability and validity of a patient self-administered daily questionnaire to assess impact of oral mucositis (OM) on pain and daily functioning in patients undergoing autologous hematopoietic stem cell transplantation (HSCT)

Stiff

Erder²,

Bensinger

et al. 2006

Bone Marrow Transplant

108

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Oral mucositis (OM) is a frequent complication of myeloablative therapy and HSCT. We evaluated the feasibility, reliability, and validity of a new patient selfreported daily questionnaire on OM and its impact on daily functions. This OM Daily Questionnaire (OMDQ), containing 10 items, was developed for use in palifermin clinical trials. In a phase 3 study, 212 patients received palifermin or placebo for three consecutive days before conditioning and three consecutive days after HSCT. Compliance rates were consistently 480% for most patients. Mouth and throat soreness (MTS) and MTSActivity Limitations (MTS-AL) (swallowing, drinking, eating, talking, and sleeping) scores on consecutive days were highly correlated (days 7,8 ¼ 0.70-0.86; test-retest reliability). Correlations among items measuring the same construct ranged between 0.5 and 0.8 (internal consistency reliability). The WHO Oral Toxicity scale was the clinical comparator to assess the criterion, discriminative, and evaluative validities of MTS-related questions. Most correlation coefficients between the WHO and MTS ranged between 0.45 and 0.55. Patients with more severe WHO OM grades had higher MTS mean scores. Changes in MTS scores were similar, but patients detected changes 1-3 days earlier than clinicians. In conclusion, the OMDQ is a feasible, reliable, valid, and responsive patient-reported measure of OM severity.

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Section: Discussionmentioning

confidence: 99%

Section: Oral Mucositis Daily Questionnairementioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

Reliability and validity of a patient self-administered daily questionnaire to assess impact of oral mucositis (OM) on pain and daily functioning in patients undergoing autologous hematopoietic stem cell transplantation (HSCT)

Stiff

Erder²,

Bensinger

et al. 2006

Bone Marrow Transplant

108

View full text Add to dashboard Cite

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“…5 Moreover, OM may prolong hospitalization, increase the use of antibiotics and other anti-infections agents, analgesics, total parenteral nutrition (TPN), and thus have significant economic costs. 6,7 Currently, there are no universal protocols that have been accepted as a standard to prevent and treat OM in the transplant setting. Research is proceeding on several new fronts, including cytokines and growth factors.…”

Section: Introductionmentioning

confidence: 99%

The influence of palifermin (Kepivance) on oral mucositis and acute graft versus host disease in patients with hematological diseases undergoing hematopoietic stem cell transplant

Nasiłowska-Adamska

Rzepecki

Mańko

et al. 2007

Bone Marrow Transplant

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In this multicenter study, we assessed the use of palifermin (recombinant human-keratinocyte growth factor 1) in the prevention of oral mucositis (OM) and acute GvHD (aGvHD) induced by a hematopoietic stem cell transplant (HSCT). Fifty-three patients with hematological diseases received three doses of palifermin (60 lg/kg once daily i.v.) pre-and post-conditioning regimens (total six doses). A retrospective control group of 53 transplant patients received no palifermin. There was a significant reduction in the incidence of OM of WHO (World Health Organization) grades 1-4 (58 vs 94%, Po0.001), 3-4 (13 vs 43%, Po0.001) and the median duration of OM (4 vs 9 days, Po0.001) in the palifermin group compared to the control group. The incidence of analgesics (32 vs 75.5%, Po0.001), opioid analgesics (24 vs 64%, Po0.001) and total parenteral nutrition (11 vs 45%, Po0.001) was also significantly reduced. The analysis of distribution of affected organs revealed that aGvHD was less prevalent in the palifermin group (P ¼ 0.036). There was no significant difference in the onset of any OM after HSCT, time to engraftment and length of hospitalization between groups. The drug was generally well tolerated and safe. Our results suggest that the use of palifermin reduces OM and probably aGvHD after HSCT, but a randomized trial is needed.

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Effect of Doxepin Mouthwash or Diphenhydramine-Lidocaine-Antacid Mouthwash vs Placebo on Radiotherapy-Related Oral Mucositis Pain

Sio

Le‐Rademacher

Leenstra

et al. 2019

JAMA

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Importance: Oral mucositis causes substantial morbidity during head-and-neck radiotherapy. In a randomized study, doxepin mouthwash was shown to reduce oral mucositis–related pain. A common mouthwash comprising diphenhydramine-lidocaine-antacid is also widely used. Objective: To evaluate the effect of doxepin or diphenhydramine-lidocaine-antacid mouthwash for the treatment of oral mucositis–related pain. Design, Settings, and Participants: A multi-institutional, 3-group, randomized, double-blind, placebo-controlled phase III trial was conducted from November 1, 2014, to May 16, 2016, in 30 institutions in the United States; 275 patients undergoing definitive head-and-neck radiotherapy with oral mucositis pain rated ≥4 (scale, 0-10) were eligible and randomized. Patients were followed up for a maximum of 28 days after randomization. The last follow-up occurred in June 2016. Interventions: Patients were randomized to doxepin (25 mg/5 mL water), 92 patients; diphenhydramine-lidocaine-antacid mouthwash, 91 patients; and placebo, 92 patients. Main Outcome and Measures: Primary end point was total oral mucositis pain reduction (area under the curve, adjusting for baseline pain scale) over 4 hours after a single dose of doxepin or diphenhydramine-lidocaine-antacid, compared with a single dose of placebo. Minimal clinically important difference was 3.5-point change. Secondary end points included drowsiness, unpleasant taste, and stinging/burning. All scales ranged from 0 (best) to 10 (worst). Results: Among the 275 patients randomized (median age, 61 years; 58 [21%] women), 227 (83%) completed treatment per protocol. Mucositis pain over the first 4 hours decreased by 11.6 points in the doxepin group, by 11.7 points in the diphenhydramine-lidocaine-antacid group, and by 8.7 points in the placebo group, with between-group differences of 2.9 points (95% CI [0.2 to 6.0]; P=.021) for doxepin vs placebo and 3.0 points (95% CI [0.1 to 5.9]; P=.004) for diphenhydramine-lidocaine-antacid vs placebo. More drowsiness (2.6 points; 95% CI [−0.7 to 5.9]; P=.03), unpleasant taste (2.8 points; 95% CI [0.8 to 4.8]; P=.002), and stinging/burning (4.9 points; 95% CI [2.9 to 6.9]; P<.001) were reported with doxepin mouthwash vs placebo. Maximum grade 3 adverse events for the doxepin mouthwash, diphenhydramine-lidocaine-antacid mouthwash, and placebo groups occurred in 3 (4%), 3 (4%), and 2 (2%) patients, respectively. Fatigue was reported by 5 patients (6%) in the doxepin mouthwash group and no patients in the diphenhydramine-lidocaine-antacid group. Conclusions and Relevance: Among patients undergoing head-and-neck radiotherapy, the use of doxepin mouthwash or diphenhydramine-lidocaine-antacid mouthwash compared with placebo significantly reduced oral mucositis pain for the first 4 hours after administration; however, the effect size was less than the minimal clinically important difference. Further research is needed to assess longer-term efficacy and safety for both mouthwashes. Trial Registration: clinicaltrials.gov Ident...

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Defining Clinically Meaningful Outcomes in the Evaluation of New Treatments for Oral Mucositis: Oral Mucositis Patient Provider Advisory Board

Cited by 59 publications

References 31 publications

Reliability and validity of a patient self-administered daily questionnaire to assess impact of oral mucositis (OM) on pain and daily functioning in patients undergoing autologous hematopoietic stem cell transplantation (HSCT)

Reliability and validity of a patient self-administered daily questionnaire to assess impact of oral mucositis (OM) on pain and daily functioning in patients undergoing autologous hematopoietic stem cell transplantation (HSCT)

The influence of palifermin (Kepivance) on oral mucositis and acute graft versus host disease in patients with hematological diseases undergoing hematopoietic stem cell transplant

Effect of Doxepin Mouthwash or Diphenhydramine-Lidocaine-Antacid Mouthwash vs Placebo on Radiotherapy-Related Oral Mucositis Pain

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