Pegfilgrastim: A Quick Look at Its R&D Progress and Clinical Results from the 2024 ASCO_GI

The original FOLFIRINOX regimen (5-fluorouracil, leucovorin, irinotecan, and oxaliplatin) for pancreatic ductal adenocarcinoma (PDAC) is associated with a high risk of febrile neutropenia (FN), and pegfilgrastim has been used to reduce the risk. However, it remains unclear how long pegfilgrastim needs to continue. On 18 Jan 2024, the phase II study of the safety and efficacy of discontinuing pegfilgrastim for pancreatic adenocarcinoma treated with FOLFIRINOX was reported in 2024 ASCO_GI.

Pegfilgrastim's R&D Progress

Pegfilgrastim, developed by Amgen, Inc., is a drug classified as a colony-stimulating factor. It targets the CSF-3R receptor and is used in the treatment of various diseases across multiple therapeutic areas. These areas include infectious diseases, hemic and lymphatic diseases, other diseases, neoplasms, immune system diseases, and cardiovascular diseases.

According to the Patsnap Synapse, Pegfilgrastim has achieved approval in several countries globally. Its highest phase of development in China is currently pending. And the clinical trial distributions for Pegfilgrastim are primarily in the United States, China and France. The key indication is Breast Cancer.

Detailed Clinical Result of Pegfilgrastim

This Non-Randomized, Single Group Assignment clinical trial (JPRN-UMIN000028055) was aimed to evaluate the safety and efficacy of FOLFIRINOX after discontinuing pegfilgrastim in PDAC patients.  

In this study, patients with PDAC who received the first three courses of FOLFIRINOX, with primary pegfilgrastim prophylaxis for FN, and did not experience FN were included. The patients continued on FOLFIRINOX without pegfilgrastim from the fourth course. The primary endpoint was development of FN. The secondary endpoints included relative dose intensity (RDI), objective response rate (ORR), progression-free survival (PFS), and overall survival (OS). The threshold and expected development of FN were 20% and 7%, respectively, at a one-sided significance level of 0.10 and statistical power of 80%. Based on this hypothesis, we calculated the number of patients needed to be 33.

The result showed that in total, 34 patients (20 male, median age 66 years) were enrolled from August 2017 to September 2021. Twenty-three patients were unresectable, and 11 were borderline resectable. FN developed in one patient. Grade 4 neutropenia was observed in six patients, grade 3 neutropenia in 10 (29.4%), anemia in four (11.8%), diarrhea in two (5.9%), and malaise, lung infection, peripheral sensory neuropathy, colitis, and biliary tract infection in one (2.9%) each. Twenty-two patients required dose reduction, mainly because of neutropenia. Mean RDIs at the sixth course of oxaliplatin, irinotecan, leucovorin, acute infusion of fluorouracil, and continuous infusion of fluorouracil were 77.0%, 88.2%, 81.8%, 84.4%, and 87.6%, respectively. ORR, median PFS, and median OS were 44.1%, 15.8 months, and 25.8 months, respectively.

It can be concluded that FOLFIRINOX can be continued safely with a low incidence of FN after discontinuing pegfilgrastim in PDAC patients who did not experience FN during the first three courses of FOLFIRINOX, if the drug dose is reduced appropriately for neutropenia from the fourth course.

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