RECITE Trial: Romiplostim Prevents Chemotherapy Side Effects and Preserves Treatment Intensity

Results from the phase 3 RECITE trial show that romiplostim significantly reduces chemotherapy-induced thrombocytopenia, enabling 84% of patients to receive chemotherapy without dose modification. In 165 patients with gastrointestinal cancers, the drug reduced chemotherapy dose modifications more than tenfold, potentially preserving treatment intensity and improving outcomes.

The RECITE trial, led by investigators at Mass General Brigham and published in the New England Journal of Medicine, suggests that romiplostim may offer an effective strategy to prevent complications and help patients maintain planned chemotherapy treatment.

Addressing an Unmet Clinical Need

Chemotherapy-induced thrombocytopenia (CIT) is a common and clinically significant complication of cytotoxic cancer therapy. By suppressing bone marrow function and impairing platelet production, chemotherapy can lead to marked reductions in platelet counts, increasing the risk of bleeding. In many cases, this forces clinicians to delay treatment or reduce chemotherapy dose intensity, potentially compromising overall treatment effectiveness. Currently, no widely available therapies are specifically approved to prevent CIT.

Lead investigator Dr. Hanny Al-Samkari, a hematologist at the Mass General Brigham Cancer Institute, emphasized the clinical importance of addressing this gap. “This work has been nearly a decade in the making, and it is so important because there are no available approved medications for chemotherapy-induced thrombocytopenia, which drastically increases a patient’s risk of major or life-threatening bleeding,” he said.

Dr. Al-Samkari, also highlighted the downstream consequences of treatment interruptions: “In an effort to prevent life-threatening bleeding in these patients, oncologists are forced to dose reduce and delay chemotherapy, often repeatedly. We know from other studies that this chemotherapy intensity reduction results in worsened outcomes of cancer treatment, including reduced overall survival and lower chance of cancer cure. Therefore, we hope that romiplostim’s ability to allow administration of full-dose chemotherapy delivered on time will translate into longer survival for patients.”

Trial Design and Patient Population

The international, double-blind, randomized, placebo-controlled RECITE study enrolled 165 patients with persistent CIT and gastrointestinal cancers who were receiving oxaliplatin-based chemotherapy. Participants had platelet counts of 85×10⁹ per liter or lower at enrollment. Patients were randomized in a 2:1 ratio to receive romiplostim or placebo over three chemotherapy cycles.

Among enrolled participants, 75% had colorectal cancer, 13% gastroesophageal cancer, and 12% pancreatic cancer. Advanced disease was common, with stage IV cancer present in 72% of patients in the romiplostim group and 61% in the placebo group. The primary endpoint was the absence of CIT-induced chemotherapy dose modifications, including dose reduction, delay, omission, or discontinuation during the second and third treatment cycles.

Significant Reduction in Chemotherapy Dose Modifications

The results demonstrated a clear benefit for patients receiving romiplostim. Chemotherapy was delivered without CIT-related modifications in 84% of patients treated with romiplostim compared with 36% in the placebo group. This corresponded to an odds ratio of 10.16 and a risk ratio of 2.77, both statistically significant.

These findings indicate that romiplostim substantially reduced the likelihood that thrombocytopenia would interfere with planned chemotherapy intensity. In practical terms, patients receiving romiplostim had more than tenfold lower odds of requiring chemotherapy dose reduction due to CIT.

Safety Profile and Adverse Events

Adverse events of grade 3 or higher occurred in 37% of patients in the romiplostim group and 22% in the placebo group. Investigators noted that these differences largely reflected the effects of multiagent chemotherapy and the fact that patients receiving romiplostim were able to maintain higher chemotherapy dose intensity.

Adverse events considered related to romiplostim or placebo were reported in 12 percent and 7 percent of patients respectively. The most common events were nausea, occurring in 2 percent of patients in each group, and headache, reported in 2 percent of the romiplostim group. None of these events were serious or led to death or treatment discontinuation.

Implications for Clinical Practice

The findings suggest that romiplostim may address a long-standing challenge in oncology practice by preventing chemotherapy-induced platelet suppression and allowing patients to maintain full-dose treatment. By reducing the need for dose reductions or delays, the therapy may help preserve treatment intensity, an important factor in achieving optimal cancer outcomes.