Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Analysis Group, Inc. | INDUSTRY |
Not provided
Not provided
Not provided
Not provided
BLU-285-2405 is a multi-center, synthetic control, observational and retrospective study designed to compare clinical outcomes for avapritinib compared with best available therapy for patients with AdvSM.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Patients from the BLU-285-2101 and BLU-285-2202 studies | Patients with advanced systemic mastocytosis who received treatment with avapritinib as part of the BLU-285-2101 and BLU-285-2202 studies | ||
| External Control Group | Patients with advanced systemic mastocytosis that received best available therapy |
Not provided
| Measure | Description | Time Frame |
|---|---|---|
| Comparative evaluation of overall survival (OS) between patients receiving best available therapy versus avapritinib in BU-285-2101 and BLU-285-2202 | Overall Survival defined as time from initiation of systemic treatment to death from any cause | up to 12 years |
| Measure | Description | Time Frame |
|---|---|---|
| Comparative evaluation between patients receiving best available therapy versus avapritinib of duration of treatment (DOT) | DOT, defined as the duration from initiation of line of systemic treatment to discontinuation of same line of treatment for any reason and; | Up to 12 years |
| Comparative evaluation between patients receiving best available therapy versus avapritinib and time to next treatment line (TtNTL) |
Not provided
Inclusion Criteria for Patients in the External Control Arm:
Diagnosed with AdvSM, with known subtype including SM-AHN, ASM, or MCL
Received at least one line of systemic therapy for AdvSM, which may include but not limited to regimens containing:
Midostaurin Cytoreductive therapy: cladribine, interferon alpha, azacitidine, decitabine Selective TKIs: imatinib, nilotinib, dasatinib Hydroxyurea Antibody therapy: brentuximab vedotin
Adult (≥18 years of age) at the initiation of first systemic line of therapy at the participating site
Had an index date at least 3 months prior to the start of data collection (in order to include patients with at least 3 months of follow-up after index date), unless date of death occurred less than three months from index date
Had an approved waiver of informed consent or signed informed consent for participation in the retrospective chart review study, if no institutional waiver from the site was granted
Exclusion Criteria for Patients in the External Control Arm
Malignancy that is not in remission at time of SM diagnosis, or new non-hematological malignancy diagnosed after SM diagnosis, except for: completely resected basal cell and squamous cell skin cancer, curatively treated localized prostate cancer, and completely resected carcinoma in situ of any site
Among patients with SM-AHN, presence of either of the following:
Received avapritinib as the first line of systemic therapy for AdvSM at participating site, or prior to initiation of first systemic therapy at participating site.
Not provided
Not provided
Not provided
Patients age 18 or older with a diagnosis of advanced SM.
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Standford Cancer Center | Palo Alto | California | 94304 | United States | ||
| Dana Farber Cancer Institute |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35790816 | Derived | Reiter A, Gotlib J, Alvarez-Twose I, Radia DH, Lubke J, Bobbili PJ, Wang A, Norregaard C, Dimitrijevic S, Sullivan E, Louie-Gao M, Schwaab J, Galinsky IA, Perkins C, Sperr WR, Sriskandarajah P, Chin A, Sendhil SR, Duh MS, Valent P, DeAngelo DJ. Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis. Leukemia. 2022 Aug;36(8):2108-2120. doi: 10.1038/s41375-022-01615-z. Epub 2022 Jul 5. |
Not provided
Not provided
IPD will not be shared
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
TtNTL, defined as the time from initiation of the line of systemic treatment to the initiation of the next line of treatment |
| Up to 12 years |
| Comparative evaluation of change in serum tryptase concentration in patients receiving best available therapy versus avapritinib | Change in serum tryptase concentration, defined as change in serum tryptase from baseline, for each line of therapy | Up to 12 years |
| To characterize the safety profile and conduct comparative evaluation of safety between patients receiving best available therapy vs. avapritinib | AEs that result in treatment modification or discontinuation, hospitalization, or death according to evaluation of responsible physician | Up to 12 years |
| Boston |
| Massachusetts |
| 02215 |
| United States |
| Medizinische Universitat Wien | Vienna | Austria |
| Universitatmedizin Mannheim | Mannheim | Baden-Wurttemberg | Germany |
| Hospital Virgen del Valle | Toledo | Spain |
| Guy's and St. Thomas' NHS Foundation Trust | London | England | United Kingdom |
| ID | Term |
|---|---|
| D034721 | Mastocytosis, Systemic |
| D007946 | Leukemia, Mast-Cell |
| ID | Term |
|---|---|
| D008415 | Mastocytosis |
| D009372 | Neoplasms, Connective Tissue |
| D018204 | Neoplasms, Connective and Soft Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D000090362 | Mast Cell Activation Disorders |
| D007154 | Immune System Diseases |
| D007938 | Leukemia |
| D015470 | Leukemia, Myeloid, Acute |
| D007951 | Leukemia, Myeloid |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
Not provided
Not provided