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Open research questions in Multiple Myeloma Research and Treatments

599 gap statements mined from Multiple Myeloma Research and Treatments papers in our 7.9M-paper local library, which holds 2,724 papers on the topic — drawn mostly from each paper's own stated research gap, future-work, challenge and limitation notes, and its abstract. The ones listed below are a selection still marked open; each names the study that raised it, with a DOI link where the paper has one.

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What the literature leaves open

  • The role of salvage autologous stem cell transplantation in relapsed/refractory multiple myeloma is not well established in the era of novel immunotherapies. There is a need to evaluate the long-term efficacy and safety of salvage ASCT in a real-world setting.

    Salvage Autologous Stem Cell Transplantation in Relapsed/Refractory Multiple Myeloma: Long-Term Efficacy and Safety Outcomes from a Single-Center Real-World Cohort · 2026 · DOI

  • The optimal dosing strategy for melphalan conditioning continues to be debated. The impact of infused CD34⁺ cell dose on engraftment kinetics and transfusion requirements is not well understood.

    Impact of Melphalan Conditioning Dose and Infused CD34⁺ Cell Dose on Engraftment Kinetics, Transfusion Requirements, and Early Transplant Outcomes Following Autologous Stem Cell Transplantation for Multiple Myeloma · 2026 · DOI

  • The study also faces challenges in terms of limited data on start and stop of maintenance. The study must account for these challenges through the use of a 6-month landmark analysis and other statistical methods.

    Impact of maintenance therapy on survival after autologous stem cell transplant for multiple myeloma regardless of response: a global analysis · 2026 · DOI

  • The optimal maintenance therapy for multiple myeloma after AHCT is not well established. The use of maintenance therapy varies globally, and its impact on overall survival and progression-free survival is not well understood. There is a need for a global analysis of the impact of different maintenance therapies on survival after AHCT for multiple myeloma.

    Impact of maintenance therapy on survival after autologous stem cell transplant for multiple myeloma regardless of response: a global analysis · 2026 · DOI

  • The study faces the challenge of limited longitudinal data. The study acknowledges the challenge of confirming the clinical relevance of the findings in prospective studies. The study notes the challenge of achieving a satisfactory response to treatment in patients with PCM.

    Diagnostic and prognostic value of bone metabolism biomarkers in newly diagnosed plasma cell myeloma · 2026 · DOI

  • Longitudinal collection of bone marrow samples across the course of MM remains challenging. Adult BM recapitulation is still challenging. Incorporating patient-derived cells into 3D models to improve accuracy is a technical challenge.

    Multiple myeloma bone disease models with integrated bone marrow adipose tissue for advancing personalized medicine · 2026 · DOI

  • Anti‐cluster of differentiation 38 (Anti‐CD38) monoclonal antibodies such as daratumumab are widely used in multiple myeloma (MM), yet the timing and magnitude of infectious risk remain incompletely defined.

    Temporal trends in infectious risk among multiple myeloma patients treated with daratumumab: A meta‐analysis · 2026 · DOI

  • Resistance to pomalidomide has been attributed to multiple mechanisms, including CRBN downregulation and activation of pro-survival signaling pathways such as NF-κB; however, the contribution of epigenetic regulators, particularly the STAT3EZH-STAT3 axis, remains insufficiently explored.

    EZH2-dependent STAT3 stabilization drives pomalidomide resistance and is targetable by EZH2 inhibition in multiple myeloma · 2026 · DOI

  • Patients and caregivers believed that prognostic knowledge was important and helpful, yet many received insufficient information.

    Patient and caregiver understanding of multiple myeloma: A scoping review · 2025 · DOI

  • However, it remains unclear if high CTC counts represent a mere surrogate of tumor burden or might reflect a distinct genomic or transcriptomic entity.

    Elevated circulating tumor cells reflect high proliferation and genomic complexity in multiple myeloma · 2025 · DOI

  • In multiple myeloma (MM), minimal residual disease (MRD) is an established endpoint for accelerated drug approval but is limited by a need for serial invasive bone marrow (BM) biopsies, which may under‐sample spatial heterogeneity and result in false negativity.

    Whole Genome Sequencing of Cell-free DNA for Assessment of Minimal Residual Disease in High-Risk Smoldering Multiple Myeloma · 2025 · DOI

  • However, the extent to which FLCs are removed by blood purification therapy remains unclear.

    Evaluation of Light Chain Removal Rates in Blood Purification Therapy for Light Chain Cast Nephropathy · 2025 · DOI

  • Although SLONM frequently occurs with monoclonal gammopathy of undetermined significance (MGUS), the coexistence with other MGUS-related disorders has not been reported.

    Monoclonal Gammopathy – the common denominator of sporadic late-onset nemaline myopathy and paraproteinemic neuropathy · 2025 · DOI

  • Given a lack of population-based screening for MGUS and its asymptomatic nature, the epidemiology of MGUS remains unknown.

    Asymptomatic Incidence of Monoclonal Gammopathy of Undetermined Significance and Preclinical Duration to Myeloma Diagnosis: A Modeling Study · 2024 · DOI

  • Additionally, data on CTPC in NDMM patients not treated with transplant are scarce.

    Circulating tumor plasma cells and peripheral blood measurable residual disease assessment in multiple myeloma patients not planned for upfront transplant · 2024 · DOI

  • Therefore future studies are warranted to define the impact of being ultra-frail on prognosis.

    Assessing frailty in myeloma: The pursuit of simplicity may sacrifice precision of predicting clinical outcomes · 2024 · DOI

  • While both the IMWG-FI and the Simplified-FI categorize patients as fit, intermediate-fit, or frail, the exact alignment of these groups remains uncertain.

    Assessing frailty in myeloma: The pursuit of simplicity may sacrifice precision of predicting clinical outcomes · 2024 · DOI

  • However, its association with disease stage, genetic alterations, and outcome has not been systematically investigated in large cohorts.

    CDK6 protein expression is associated with disease progression and treatment resistance in multiple myeloma · 2024 · DOI

  • The study had a retrospective design. The study had limited longitudinal data. The sample size was relatively small. The study only involved patients with newly diagnosed PCM.

    Diagnostic and prognostic value of bone metabolism biomarkers in newly diagnosed plasma cell myeloma · 2026 · DOI

  • Sparse research had a primary aim of investigating understanding of MM.

    Patient and caregiver understanding of multiple myeloma: A scoping review · 2025 · DOI

  • Despite hematologic remission, renal recovery was limited by a high number of casts, severe interstitial fibrosis and tubular atrophy, and the delay in treatment initiation.

    Evaluation of Light Chain Removal Rates in Blood Purification Therapy for Light Chain Cast Nephropathy · 2025 · DOI

  • Despite differing clinical courses, shared pathology suggests common mechanisms, with mitochondrial abnormalities as a possibly under-recognized feature.

    Monoclonal Gammopathy – the common denominator of sporadic late-onset nemaline myopathy and paraproteinemic neuropathy · 2025 · DOI

  • Potentially important single-nucleotide polymorphisms (SNPs) in the genes coding for IKZF1 and IKZF3 have been identified, but they have not been analyzed in MM patients before.

    Polymorphisms within genes encoding Ikaros family proteins IKZF1 and IKZF3 in multiple myeloma patients treated with thalidomide · 2024 · DOI

  • Furthermore, the incidence of MGUS is poorly understood; a better understanding of its epidemiology will be critical in designing appropriate screening guidelines once an effective treatment has been developed.

    The Undetermined Significance of Screening for Monoclonal Gammopathy of Undetermined Significance · 2024 · DOI

  • Prospective evaluation of salvage ASCT compared to contemporary immunotherapies, - Investigation of prognostic factors for PFS and OS after salvage…

    Salvage Autologous Stem Cell Transplantation in Relapsed/Refractory Multiple Myeloma: Long-Term Efficacy and Safety Outcomes from a Single-Center Real-World Cohort · 2026 · DOI

Most-cited papers in Multiple Myeloma Research and Treatments

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Open questions raised by papers listed on this page include: (1) The role of salvage autologous stem cell transplantation in relapsed/refractory multiple myeloma is not well established in the era of novel immunotherapies (“Salvage Autologous Stem Cell Transplantation in Relapsed/Refractory Multiple…”, 2026); (2) The impact of infused CD34⁺ cell dose on engraftment kinetics and transfusion requirements is not well understood (“Impact of Melphalan Conditioning Dose and Infused CD34⁺ Cell Dose on…”, 2026); (3) The optimal maintenance therapy for multiple myeloma after AHCT is not well established (“Impact of maintenance therapy on survival after autologous stem cell transplant…”, 2026); (4) It remains unclear if high CTC counts represent a mere surrogate of tumor burden or might reflect a distinct genomic or transcriptomic entity (“Elevated circulating tumor cells reflect high proliferation and genomic…”, 2025); (5) The extent to which FLCs are removed by blood purification therapy remains unclear (“Evaluation of Light Chain Removal Rates in Blood Purification Therapy for Light…”, 2025). 599 gap statements have been mined from Multiple Myeloma Research and Treatments papers in our 7.9M-paper local library, which holds 2,724 papers on the topic; the gaps come from whichever of those papers state one. They are mostly the research gaps the authors state and the papers' abstracts, plus future-work, limitations and challenges passages. The ones listed below are a selection still marked open; each names the study that raised it, with a DOI link where the paper has one, so you can read the original claim in context.

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