BRAF V600 Mutation Positive Melanoma
Conditions
Brief summary
This was a multi-center, observational, retrospective cohort study to evaluate the effectiveness and safety of dabrafenib in combination with trametinib in Chinese patients with unresectable or metastatic BRAF V600 mutation positive melanoma, for mucosal melanoma patients (Cohort A) and non-mucosal melanoma patients (Cohort B, cutaneous and acral melanoma), separately. Study population was identified as patients initiating dabrafenib plus trametinib from 01 May 2020 to 31 July 2022 who fulfilled the inclusion/exclusion criteria. The follow-up period ended at the earliest of the following: end of study observation period (i.e., 31 December 2022), death, upon withdrawal of consent or the last available record.
Interventions
None listed
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Cohorts A and B: * Initiated dabrafenib and trametinib combination therapy (D+T) according to approved label between 01 May 2020 and 31 July 2022 * Was ≥18 years old of age at the initiation of D+T * Had at least one tumor assessment after initiation of D+T * Written informed consent if requested by the study site Cohort A Only • Confirmed BRAF V600 mutation positive mucosal melanoma that was unresectable or metastatic Cohort B Only • Confirmed BRAF V600 mutation positive non-mucosal melanoma (cutaneous and acral melanoma) that was unresectable or metastatic
Exclusion criteria
None specified
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Real-world overall response rate (rwORR) | Up to approximately 2.6 years | ORR was defined as the percentage of patients demonstrating a best overall response as complete response (CR) or partial response (PR) by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1, or as documented per physician assessment, as available. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of patients per sex | Baseline | — |
| Number of years of disease history at treatment initiation since initial melanoma diagnosis | Baseline | — |
| Number and percentage of patients per anatomic sites of origin | Baseline | — |
| Number of years of disease history at treatment initiation since unresectable or metastatic melanoma diagnosis | Baseline | — |
| Number and percentage of patients per tumor stage | Baseline | — |
| Number and percentage of patients with occurrence of tumor metastasis | Baseline | — |
| Number and percentage of patients per metastatic location | Baseline | — |
| Number and percentage of patients per metastases | Baseline | — |
| Lactate dehydrogenase (LDH) levels | Baseline | — |
| Eastern Cooperative Oncology Group (ECOG) performance status | Baseline | ECOG performance status describes a patient's level of functioning in terms of their ability to care for themself, daily activity, and physical ability (walking, working, etc.). Scores can range from a lower value of 0 (fully active, able to carry on all pre-disease performance without restriction) up to 5 (dead). |
| Number and percentage of patients who had at least one surgery for melanoma prior to D+T treatment | Baseline | — |
| Number and percentage of patients per type of surgery | Baseline | — |
| Number and percentage of patients per name of surgery | Baseline | — |
| Number and percentage of patients per surgical and medical procedure | Baseline | — |
| Number and percentage of patients who had at least one anti-neoplastic drug for melanoma prior to D+T treatment | Baseline | — |
| Number and percentage of patients with prior anti-neoplastic drugs for melanoma per treatment intent | Baseline | — |
| Number and percentage of patients with prior anti-neoplastic drug for melanoma per treatment setting | Baseline | — |
| Number and percentage of patients with prior anti-neoplastic drug for melanoma per line of treatment | Baseline | — |
| Number and percentage of patients with prior anti-neoplastic drug for melanoma per treatment type | Baseline | — |
| Number and percentage of patients with systemic anti-neoplastic treatment after D+T per line of treatment | Up to approximately 2.6 years | — |
| Number and percentage of patients per reason for immunotherapy discontinuation | Baseline | — |
| Number and percentage of patients with prior anti-neoplastic drug for melanoma with best overall tumor response | Baseline | — |
| Number and percentage of patients with systemic anti-neoplastic treatment after D+T and treatment ongoing at end of follow up | Up to approximately 2.6 years | — |
| Number and percentage of patients per prior anti-neoplastic drug for melanoma | Baseline | — |
| Number and percentage of patients who had at least one radiotherapy for melanoma prior to D+T treatment | Baseline | — |
| Number and percentage of patients with prior radiotherapy for melanoma per treatment intent | Baseline | — |
| Number and percentage of patients with prior radiotherapy for melanoma per treatment setting | Baseline | — |
| Number and percentage of patients per radiation site | Baseline | — |
| Mean total dosage for all radiotherapy | Baseline | — |
| Number and percentage of patients with prior radiotherapy for melanoma with best overall tumor response | Baseline | — |
| Number and percentage of patients with dabrafenib plus trametinib treatment per line of treatment | Up to approximately 2.2 years | — |
| Number and percentage of patients with dabrafenib plus trametinib treatment per treatment intent | Up to approximately 2.2 years | — |
| Number and percentage of patients with dabrafenib plus trametinib treatment per treatment setting | Up to approximately 2.2 years | — |
| Number and percentage of patients per type of D+T treatment change | Up to approximately 2.2 years | — |
| Number and percentage of patients per reason for D+T treatment change | Up to approximately 2.2 years | — |
| Mean duration of D+T, if not ongoing to end of study follow-up | Up to approximately 2.2 years | — |
| rwORR of dabrafenib plus trametinib among non-mucosal melanoma patients (FAS) | Up to approximately 2.6 years | — |
| Number and percentage of patients with systemic anti-neoplastic treatment after D+T per treatment type | Up to approximately 2.6 years | — |
| Real-world disease control rate (rwDCR) of D+T (FAS) | Up to approximately 2.6 years | rwDCR was defined as the percentage of patients with a best overall response of complete response (CR) or partial response (PR) or stable disease (SD) or non-CR or non-progressive disease (non-PD), per RECIST version 1.1 or as documented per physician assessment, as available. |
| Real-world duration of response (rwDOR) of dabrafenib plus trametinib | Up to approximately 2.6 years | For the subset of patients with CR or PR, rwDORn was defined as the time from the date of the first documented CR or PR per RECIST version 1.1 or as documented per physician assessment as available, to the first disease progression or death due to any cause. |
| Real-world progression-free survival (rwPFS) for dabrafenib plus trametinib (FAS) | Up to approximately 2.6 years | rwPFS was defined as the time from the start of treatment to the first documented disease progression or death due to any cause. |
| Real-world overall survival (rwOS) since D+T initiation (FAS) | Up to approximately 2.6 years | rwOS was defined as the time from start of treatment to death due to any cause. |
| Time to treatment discontinuation (FAS) | Up to approximately 2.6 years | — |
| Number and percentage of patients with adverse events of special interest (AESIs) (FAS) | Up to approximately 2.6 years | AESIs were defined based on the case retrieval strategy file available at the time of analysis. |
| Number and percentage of patients with serious adverse events (SAEs) (FAS) | Up to approximately 2.6 years | — |
| rwPFS for dabrafenib plus trametinib (MMS), by immunotherapy use | Up to approximately 2.6 years | rwPFS was defined as the time from the start of treatment to the first documented disease progression or death due to any cause. |
| rwPFS for dabrafenib plus trametinib (NMS), by immunotherapy use | Up to approximately 2.6 years | rwPFS was defined as the time from the start of treatment to the first documented disease progression or death due to any cause. |
| rwOS since D+T initiation (MMS), by immunotherapy use | Up to approximately 2.6 years | rwOS was defined as the time from start of treatment to death due to any cause. |
| rwOS since D+T initiation (NMS), by immunotherapy use | Up to approximately 2.6 years | rwOS was defined as the time from start of treatment to death due to any cause. |
| Mean Age | Baseline | — |
| Number and percentage of patients with systemic anti-neoplastic treatment after D+T with best overall tumor response | Up to approximately 2.6 years | — |
| Number and percentage of patients who had systemic anti-neoplastic treatment after D+T treatment | Up to approximately 2.6 years | — |
| Number and percentage of patients who had systemic anti-neoplastic treatment after D+T treatment per medication | Up to approximately 2.6 years | — |
| Number and percentage of patients per concomitant medication | Up to approximately 2.2 years | — |
| Real-world overall survival since the first anti-neoplastic drug treatment for advanced/metastatic melanoma (FAS) | Up to approximately 2.6 years | — |
| Real-world overall survival since the first anti-neoplastic drug for advanced/metastatic melanoma (NMS), by immunotherapy use | Up to approximately 2.6 years | — |
| Number and percentage of patients with systemic anti-neoplastic treatment after D+T | Up to approximately 2.6 years | — |
Countries
China
Outcome results
None listed