Long-term survival benefits of intrathecal autologous bone marrow-derived mesenchymal stem cells (Neuronata-R®: lenzumestrocel) treatment in ALS: Propensity-score-matched control, surveillance study

ObjectiveNeuronata-R® (lenzumestrocel) is an autologous bone marrow-derived mesenchymal stem cell (BM-MSC) product, which was conditionally approved by the Korean Ministry of Food and Drug Safety (KMFDS, Republic of Korea) in 2013 for the treatment of amyotrophic lateral sclerosis (ALS). In the present study, we aimed to investigate the long-term survival benefits of treatment with intrathecal lenzumestrocel.MethodsA total of 157 participants who received lenzumestrocel and whose symptom duration was less than 2 years were included in the analysis (BM-MSC group). The survival data of placebo participants from the Pooled-Resource Open-Access ALS Clinical Trials (PROACT) database were used as the external control, and propensity score matching (PSM) was used to reduce confounding biases in baseline characteristics. Adverse events were recorded during the entire follow-up period after the first treatment.ResultsSurvival probability was significantly higher in the BM-MSC group compared to the external control group from the PROACT database (log-rank, p < 0.001). Multivariate Cox proportional hazard analysis showed a significantly lower hazard ratio for death in the BM-MSC group and indicated that multiple injections were more effective. Additionally, there were no serious adverse drug reactions found during the safety assessment, lasting a year after the first administration.ConclusionThe results of the present study showed that lenzumestrocel treatment had a long-term survival benefit in real-world ALS patients

An Effective Management of Human Resource on Organizational Learning

ABSTRAC

Comparison of Treatment Outcomes between Breast Conserving Surgery Followed by Radiotherapy and Mastectomy Alone in Patients with T1-2 Stage and 1-3 Axillary Lymph Nodes in the Era of Modern Adjuvant Systemic Treatments

<div><p>Purpose</p><p>The role of postmastectomy radiotherapy in the treatment of T1–2 primary tumor with 1–3 positive lymph nodes is controversial. We compared treatment outcomes between breast conserving surgery followed by radiotherapy (BCS+RT) and total mastectomy alone (TM) in the setting of modern adjuvant systemic treatments.</p><p>Methods</p><p>Patients with T1–2 primary breast cancer and 1–3 positive lymph nodes who were treated between 2001 and 2011 were divided into 2 groups based on the treatment approach: BCS+RT (<i>n</i> = 169) and TM (<i>n</i> = 117). All patients received adjuvant chemotherapy including taxanes. Adjuvant endocrine therapy was administered to patients with positive hormone receptors according to their menstrual status.</p><p>Results</p><p>During a median follow-up of 76.5 months, 21 patients (7.3%) experienced locoregional recurrence as the first event, including 7 patients (4.1%) in the BCS+RT group and 14 patients (12.0%) in the TM group. The 5-year cumulative incidence rate of locoregional recurrence was 2.5% for BCS+RT versus 9.5% for TM (<i>p</i> = 0.016). Competing risk regression analysis revealed that TM was associated with a relative risk for locoregional recurrence of 5.347 (<i>p</i> = 0.003). TM was also associated with a significantly lower 5-year disease-free survival rate compared with BCS+RT (hazard ratio, 2.024; 95% confidence interval, 1.090–3.759; <i>p</i> = 0.026).</p><p>Conclusion</p><p>To improve treatment outcomes for TM even after modern systemic treatments, postmastectomy radiotherapy might be required for patients with T1–2 primary breast cancer and 1–3 positive lymph nodes.</p></div

Multivariate analysis for the disease-free survival rate.

<p>Multivariate analysis for the disease-free survival rate.</p

Patient characteristics.

<p>Patient characteristics.</p

Competing risk analysis for the cumulative incidence rate of locoregional recurrence.

<p>Competing risk analysis for the cumulative incidence rate of locoregional recurrence.</p

Comparison of the 5-year cumulative incidence rate of locoregional recurrence between the breast-conserving surgery plus radiotherapy (solid line) and total mastectomy alone groups (dotted line).

<p>Comparison of the 5-year cumulative incidence rate of locoregional recurrence between the breast-conserving surgery plus radiotherapy (solid line) and total mastectomy alone groups (dotted line).</p

Comparison of the 5-year disease-free survival rate between the breast-conserving surgery plus radiotherapy (solid line) and total mastectomy groups (dotted line).

<p>Comparison of the 5-year disease-free survival rate between the breast-conserving surgery plus radiotherapy (solid line) and total mastectomy groups (dotted line).</p