<p>Improvements in primary central nervous system lymphoma (PCNSL) treatment outcomes over the past several decades reflect advances in therapeutic strategies. However, real-world challenges impact both treatment goals and outcomes, with presentations at the recent <strong>67th American Society of Hematology (ASH) Annual Meeting and Exposition</strong> offering real-world insights into these topics.</p> <p><br></p> <p><em>Following these presentations, featured expert Matthias Holdhoff, MD, PhD, was interviewed by </em>Conference Reporter<em> Medical Director Lauren Weinand, MD. Clinical perspectives from Dr Holdhoff on these findings are presented here.</em></p>

The first question we ask when establishing treatment goals for a patient with PCNSL is whether they are candidates for high-dose methotrexate–based induction therapy, an aggressive approach aimed at achieving a complete response. Then, the second question is whether they would be suitable for consolidation with high-dose chemotherapy and autologous stem cell transplant (ASCT). However, other treatment approaches are now also available for patients who do not meet the criteria for this aggressive treatment approach. Data from the 67th ASH Annual Meeting and Exposition help advance our knowledge on PCNSL treatment goals and real-world outcomes.

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For example, Ayo Falade, MD, MBA, et al presented real-world evidence on treatment patterns and survival outcomes in PCNSL using data from the National Cancer Database (NCDB) for patients who received a diagnosis between 2004 and 2020 (abstract 4494). The data are not very granular, but they provide the big picture of overall survival (OS) in patients with PCNSL based on treatment. This was a very large patient cohort (ie, 13,390 patients with survival data) with a median age at diagnosis of 67 years, which is representative of this patient population.

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Patients who were treated with high-dose chemotherapy followed by ASCT had the longest median OS (mOS), which was not reached, while individuals who received radiation alone had the poorest outcomes (mOS, 3.9 months). Patients who received chemoradiation had an mOS of 31.8 months, and those who received high-dose chemotherapy alone had an mOS of 13.9 months. Older age, comorbidities, and African American race were associated with inferior outcomes, while more recent year of diagnosis, treatment at an academic center or a high case-volume center, and treatment type (ie, high-dose chemotherapy followed by ASCT, chemoradiation, and multiagent chemotherapy) were predictors of superior OS.

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In this study, there was clear superiority for patients treated with high-dose chemotherapy (eg, high-dose methotrexate–based therapy) and ASCT. This is consistent with current clinical opinion and with data from other individual studies. Of course, these treatments require a center that can offer such options to patients. Overall, I think that this study provides a gut check for which treatment modalities might be the most effective for patients with PCNSL, despite the study’s limitations.

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Another real-world evidence presentation at this year’s ASH meeting by Ysaith Orellana Ascencio, DO, evaluated how treatment facility type and sociodemographic factors influence treatment patterns and survival in patients with PCNSL (abstract 4493). This study also used data from the NCDB for patients who received a diagnosis between 2004 and 2022. The authors reported that patients do better when treated at academic centers or centers of excellence for PCNSL, an idea that is already supported in clinical guidelines. mOS was significantly longer at academic centers than at community practices (19.6 vs 10.5 months, respectively). Additionally, 30- and 90-day mortality were lower at academic centers compared with community practices (2.2% vs 2.8% and 6.4% vs 8.6%, respectively). Finally, long-term survival was superior at academic centers, with 2-, 5-, and 10-year OS rates of 38.9%, 31%, and 24.8%, respectively, vs 30.6%, 24.1%, and 18.9%, respectively, at community practices.

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I think that these data are very relevant to clinical practice. They reinforce the idea that referrals to centers with subspecialty expertise in PCNSL, such as large academic institutions, should be strongly considered for this rare disease for which effective therapies and promising clinical trial options exist.