Patient Preferences on Clinical Decision Making in Multiple Myeloma.

Purpose: To better understand the priorities that guide patients with multiple myeloma, we surveyed patients on four different treatment scenarios, each of treatment strategies shown to improve progression-free survival (PFS) but offering similar overall survival (OS) outcomes.

Methods: We conducted a survey using the HealthTree Cure Hub by the HealthTree Foundation, the largest online portal for people with plasma cell dyscrasias.

Results: The primary analysis cohort included 466 participants with myeloma, while an additional 297 responses from patients with smoldering myeloma or monoclonal gammopathy of uncertain significance were analyzed separately.

Assessing Outcomes Emerging After Conversion to Regular Approval for Cancer Drug Indications Granted Accelerated Approval, 1992-2021.

Background: The FDA's accelerated approval pathway expedites cancer drug approvals based on surrogate measures, while clinical benefit is assessed in post-approval confirmatory trials. Many confirmatory trials also rely on surrogate measures rather than overall survival (OS) or quality of life (QoL). We evaluated how often accelerated approvals demonstrate clinical benefits at the time of conversion to regular approval, and if not, how often OS, QoL, or substantial clinical benefit emerge post-conversion.

Predictors of withdrawal of anticancer drug indications granted accelerated approval: a retrospective cohort study.

Background: The accelerated approval pathway allows the FDA to approve drugs for serious conditions based on promising surrogate measures, with confirmatory studies required later. If subsequent testing shows an unfavorable benefit-risk profile, the indication may be withdrawn. This study aimed to identify factors associated with the withdrawal of drug indications following accelerated approval.

Medicare spending and use of subcutaneous biologic formulations with hyaluronidase.

Several intravenously administered biologic drugs have been reformulated with hyaluronidase to enable subcutaneous delivery. This offers greater convenience and fewer infusion reactions while also increasing overall spending if biosimilar competition for the subcutaneous versions begins later than for the original intravenous versions. As of December 2024, at least 9 biologics had investigational or approved hyaluronidase versions.

Beyond maximum grade: tolerability of immunotherapies, cellular therapies, and targeted agents in haematological malignancies.

The increasing use of immunotherapeutic approaches, cellular therapies, and targeted agents is rapidly and profoundly changing the treatment paradigms of haematological malignancies. These novel therapies are increasingly incorporated into earlier lines of treatment. Some are administered for a fixed duration, often with curative intent, whereas others are administered chronically for disease control.

Non-relapse mortality with bispecific antibodies: A systematic review and meta-analysis in lymphoma and multiple myeloma.

Bispecific antibodies (BsAb) are associated with distinct immune-related toxicities that impact morbidity and mortality. This systematic review and meta-analysis examined non-relapse mortality (NRM) with BsAb therapy in B-cell non-Hodgkin lymphoma (NHL) and multiple myeloma (MM). A PubMed and Embase search up to October 2024 identified 29 studies (21 NHL, 8 MM) involving 2,535 patients.

Financial challenges of being on long-term, high-cost medications.

The isocitrate dehydrogenase (IDH) inhibitor, vorasidenib, may offer a promising new treatment option for patients with IDH-mutant gliomas. However, the indefinite nature of this targeted therapy raises significant financial concerns. High costs of targeted cancer therapies, often exceeding $150 000 annually, contribute to financial toxicity, characterized by medical debt, income loss, and psychological stress, and place stress on health systems.

Advancing Global Pharmacoequity in Oncology.

Importance: Limited availability and affordability of cancer drugs contribute to staggering disparities in cancer survival between high-income and low- and middle-income countries (LMICs). As infrastructure for cancer care rapidly develops, there is an urgent need to reduce prices and improve access to cancer medicines in LMICs to advance pharmacoequity globally.

Observations: Prior strategies to expand access to cancer medicines in LMICs have primarily relied on charity or differential pricing and have yielded limited results.

Second Primary Malignancies after CAR T-Cell Therapy: A Systematic Review and Meta-analysis of 5,517 Lymphoma and Myeloma Patients.

Purpose: Chimeric antigen receptor (CAR) T-cell therapy is a potent immunotherapy for hematologic malignancies, but patients can develop long-term adverse events, including second primary malignancies (SPM) that impact morbidity and mortality. To delineate the frequency and subtypes of SPMs following CAR-T in lymphoma and myeloma, we performed a systematic review and meta-analysis.

Experimental Design: A literature search was conducted in the MEDLINE, Embase, and Cochrane CENTRAL databases.

Evaluating early intervention in smoldering myeloma clinical trials: a systematic review.

Background: Smoldering multiple myeloma (SMM), an asymptomatic precursor of multiple myeloma (MM), carries a variable risk of progression to MM. There is little consensus on the efficacy or optimal timing of treatment in SMM. We systematically reviewed the landscape of all clinical trials in SMM.

A systematic review and meta-analysis of nonrelapse mortality after CAR T cell therapy.

Although chimeric antigen receptor (CAR) T cell therapy represents a transformative immunotherapy, it is also associated with distinct toxicities that contribute to morbidity and mortality. In this systematic review and meta-analysis, we searched MEDLINE, Embase and CINAHL (Cochrane) for reports of nonrelapse mortality (NRM) after CAR T cell therapy in lymphoma and multiple myeloma up to March 2024. After extraction of causes and numbers of death, we analyzed NRM point estimates using random-effect models.

Clinical Benefit and Regulatory Outcomes of Cancer Drugs Receiving Accelerated Approval.

Importance: The US Food and Drug Administration's (FDA) accelerated approval pathway allows approval of investigational drugs treating unmet medical needs based on changes to surrogate measures considered "reasonably likely" to predict clinical benefit. Postapproval clinical trials are then required to confirm whether these drugs offer clinical benefit.

Objective: To determine whether cancer drugs granted accelerated approval ultimately demonstrate clinical benefit and to evaluate the basis of conversion to regular approval.