Background Hematopoietic stem cell transplantation (HSCT) is the only curative treatment for several hematologic malignancies and bone marrow failure syndromes. Donor availability remains a critical factor influencing access to HSCT, particularly in low- and middle-income countries where unrelated donor registries are scarce. Haploidentical HSCT using post-transplant cyclophosphamide (PTCy) has expanded donor options and improved accessibility, but comparative data from Latin American centers are still underreported. Methods We performed a retrospective cohort study including all consecutive patients who underwent allogeneic HSCT between January 2020 and December 2024 at a tertiary hematology referral center in northeastern Mexico. Clinical and transplant-related variables were analyzed, including diagnosis, donor type, CD34+ cell dose, and early mortality (30 and 100 days). Survival outcomes included overall survival (OS) and relapse-free survival (RFS) were estimated using Kaplan-Meier and compared with the log-rank test (p < 0.05). Conditioning regimens were based on CyFlu plus melphalan ± TBI or busulfan; GVHD prophylaxis included PTCy, a calcineurin inhibitor, and mycophenolate mofetil. Results Among 190 transplanted patients (mean age 34.8 ± 16 years; 62.6% male), diagnoses included ALL (34.7%), AML (34.7%), aplastic anemia (14.7%), MDS (5.3%), lymphomas (7.9%), and others (2.7%). Haploidentical donors were used in 65.3% and HLA-identical donors in 34.7%. The median infused CD34+ cell dose was 9.9 × 10⁶/kg, with a median follow-up of 288 days. Early post-transplant mortality was low, with 30-day mortality rates of 6.1% in the haploidentical group and 4.5% in the identical group, and 100-day mortality of 14.5% and 13.6%, respectively. One-year OS was 68.1% for haploidentical and 79.7% for identical HSCT (p = 0.153), while RFS was 84.7% vs 81.2% (p = 0.438)(Figure 1). Subgroup analysis showed higher OS in aplastic anemia for identical HSCT (p = 0.003) and superior RFS in AML/MDS for haploidentical HSCT (p = 0.045). The incidence of grade III–IV acute GVHD was 9.1% in identical and 12.1% in haploidentical transplants, while moderate to severe chronic GVHD occurred in 12.1% and 21.0%, respectively; neither form of GVHD significantly affected overall or relapse-free survival. Conclusions Haploidentical HSCT achieved survival outcomes comparable to HLA-identical transplants, validating its role as an effective alternative when matched donors are unavailable. Disease-specific trends suggest identical HSCT remains preferable for aplastic anemia, while haploidentical strategies may offer enhanced relapse control in myeloid neoplasms. These findings support haploidentical HSCT as a feasible, cost-effective approach for expanding transplant access in resource-limited regions.
PURPOSEThis study aims to evaluate the safety, feasibility, effectiveness, and cost-saving potential of a shortened, vial-sharing outpatient blinatumomab regimen for treating relapsed or refractory (R/R) ALL in children.MATERIALS AND METHODSWe conducted a retrospective study of pediatric patients with R/R B-cell ALL (B-ALL) treated with a shortened outpatient blinatumomab regimen (<21 days), aiming to achieve a negative measurable residual disease (MRD) remission before hematopoietic stem-cell transplantation (HSCT).RESULTSTwelve patients were included: three (25%) with primary refractory disease and nine (75%) with relapse. The median follow-up time was 33 months (range, 10-76 months). Blinatumomab was administered for a median of 19 days (range, 11-21), with 75% (9 of 12) of patients completing treatment entirely on an outpatient basis. Five patients (62%) achieved CR with undetectable MRD, and all four patients who initiated treatment because of persistent MRD achieved MRD clearance (100%). Ten patients (83%) proceeded to haploidentical HSCT. The estimated 3-year overall survival (OS) was 54%, and the relapse-free survival (RFS) was 44%. These optimization measures resulted in a 43% reduction in drug-related expenditures.CONCLUSIONReduced-duration outpatient blinatumomab shows promise as a context-adapted strategy for heavily pretreated pediatric patients with ALL. Given the small retrospective cohort, these findings should be interpreted with caution.
Introduction Cerebral palsy (CP) and autism spectrum disorder (ASD), though distinct in etiology, share underlying mechanisms such as chronic inflammation, immune dysregulation, and impaired neuroplasticity. Hematopoietic and mesenchymal progenitor cells have been investigated for their immunomodulatory, anti-inflammatory, and neurotrophic properties. Additionally, they exhibit antimicrobial activity through secretion of antimicrobial peptides (Fig.1). These features suggest that autologous bone marrow–derived total nucleated cells (BM-TNCs), may provide neuroregenerative and protective effects when administered intrathecally (IT). Objectives To evaluate long-term and infection-related safety, procedure-associated symptoms, and caregiver-reported outcomes of IT administration of mobilized autologous BM-TNCs in patients with CP and ASD, based on more than 15 years of institutional experience. Methods A retrospective descriptive cohort of 1,212 patients was analyzed (Table 1). All received a single IT infusion of G-CSF–mobilized autologous BM-TNCs. Data collected included demographics, cell counts, microbiological cultures, adverse events, and Parent Reported Outcome Measures. Results Over 70% of caregivers reported improvements in at least one functional domain (attention, language, social interaction, or motor control) within 3–6 months, with sustained long-term effects. CP patients improved mainly in gross motor function, while ASD patients showed better outcomes in socialization and communication. Regarding infection safety, 36 patients (2.97%) presented positive microbiological cultures; however, no neuroinfections or systemic infection were reported. Median cell dose injected was similar between patients with positive and negative cultures, with no significant correlation between cell dose and culture positivity (p = 0.136). The most common isolates were Staphylococcus (47.3%), Micrococcus, and Bacillus. Mild, transient symptoms such as irritability, headache, nausea/vomiting, and fever were common; seizures reported in just one patient (Table 2). Conclusion IT mobilized autologous BM-TNCs demonstrated a favorable long-term safety profile and caregiver-reported improvements in CP and ASD. Findings suggest shared mechanisms of action involving immunomodulation, cytokine reduction, neuronal plasticity, and antimicrobial activity. These results support BM-TNC therapy as a safe, multifunctional approach in regenerative and translational medicine and highlight the need for multicenter studies with validated functional scales and objective biomarkers.
OBJECTIVE:To establish the relationship of minimal residual disease (MRD) status with survival outcomes after outpatient hematopoietic stem cell transplantation (HSCT) with reduced-intensity conditioning (RIC) in patients with acute lymphoblastic leukemia (ALL). MATERIAL AND METHODS:A single-center retrospective study including 145 ALL patients who received peripheral blood (PB) RIC HSCT was carried out. MRD was measured by multiparametric flow cytometry (MFC) before and within 100 days after HLA-identical or haploidentical transplantation, with >0.01% leukemic cells considered a positive MRD result. Overall survival (OS) and relapse-free survival (RFS) were estimated by the Kaplan-Meier method, associations with clinical variables were calculated by Cox regression analysis, and competing risks were determined with Fine-Gray models. RESULTS:Before transplantation, 32 (22.06%) patients were MRD positive, 16 (50%) relapsed, and 16 (50%) died, whereas 24 (16.1%) were MRD positive after HSCT, 17 (70.8%) relapsed, and 15 died (62.5%). Eleven patients were MRD positive before and after HSCT; 7 (63.6%) relapsed, and 8 (72%) died. In multivariate analysis, a higher risk for lower OS was found in patients who relapsed (hazard ratio [HR] = 5.04, P = .001) and a higher risk was found for lower RFS in those with post-HSCT MRD positive (HR = 2.11, P = .034). No differences according to acute graft-versus-host disease (GVHD) were identified, whereas chronic GVHD led to higher RFS. In univariate analysis, pre-HSCT MRD positive was associated with lower RFS. A positive MRD status after HSCT was associated with cytokine release syndrome (CRS) (HR = 4.25, P = .019), clinical stage (HR = 0.16, P = .044), and mucositis (HR = 4.45 P = .040). CONCLUSION:An MRD-positive status after hematopoietic stem cell transplantation was associated with lower survival outcomes and with CRS development.
Introduction In relapsed or refractory acute lymphoblastic leukemia (RR ALL), total marrow and lymphoid irradiation (TMLI) is an emerging alternative with a better toxicity profile than total body irradiation (TBI). We report the results of a multicenter experience with TMLI in adults with RR ALL and compare them to a chemotherapy-based historical cohort. Objetives The primary outcome was day 100 non-relapse mortality. Secondary: Graft failure, adverse events, overall survival (OS), relapse-free survival (RFS), and GVHD rates. Methods RR ALL 16-65 years conditioned with a TMLI-based regimen in two institutions were included. TMLI conditioning consisted of cyclophosphamide (Cy) 350 mg/m² and fludarabine (Flu) 25 mg/m² (-6 to -4) followed by TMLI 12 Gy (-3 to -1) in 6 fractions of 2 Gy BID, using tomotherapy. A cranial 6 Gy boost was given for prior CNS disease. The historical control group consisted of consecutive patients in the 2 years preceding the availability of TMLI. The historical cohort received CyFlu plus melphalan 140 mg/m2 plus 2 Gy of TBI for haploidentical recipients. Graft source was peripheral blood. Graft-versus-host disease (GVHD) prophylaxis was post-transplant cyclophosphamide, tacrolimus and mycophenolate mofetil. Results 22 patients with B-ALL, median age of 21 years (16-54) 59% male, and 2 prior treatment lines (1-4) received TMLI. Donors were haploidentical in 72.7%, median CD34+ dose 10 × 10⁶/kg (5.2–15). No difference to the baseline characteristics of n=21 CyFluMel patients were found, except for higher blinatumomab exposure in TMLI. Engraftment, graft failure, toxicity, severe mucositis, GVHD incidence, and NRM rates were similar. The hospitalization rate was lower with TMLI (50% vs. 76.2% p=.076), with non-infectious causes predominating in this group (social support 13.6%, oral intolerance 9.1%) vs fever and neutropenia (66.7%) and mucositis (4.8%) (p=.045) in CyFluMel. All patients achieved MRD negativity at day +60. One-year OS and RFS did not significantly differ, though OS was lower in TMLI (76.2% vs 93%; p =0.15) as 2 patients with high risk disease died due to complications of early relapse. Conclusions Myeloablative doses of TMLI have a similar safety profile to reduced intensity chemotherapy-based conditioning in adults with ALL, with lower rates of hospitalization due to infectious complications. Longer follow-up is needed to establish whether TMLI is similarly or more effective than chemotherapy in preventing relapses.
Background Previous evidence suggests that the diagnostic role of bone marrow aspiration (BMA) is altered by the hematological malignancy itself, thereby reducing its diagnostic accuracy and potentially delaying patient care. This association, however, remains largely unevaluated.Methods We used a multivariable logistic regression model to estimate the association between hematological malignancies and the quality of the BMA, defined as optimal or suboptimal. Our primary outcome evaluated the association between hematological condition type and the quality of the bone marrow aspirate. Our secondary outcomes were to determine laboratory and patient-specific risk factors associated with the quality of the bone marrow aspirate.Results We analyzed a total of 875 patients who had either acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), multiple myeloma (MM), or non-Hodgkin lymphoma (NHL). We found that patients with AML and MDS were associated with a suboptimal BMA (OR 0.72; 95% CI 0.56-0.91 and OR 0.63; 95% CI 0.50-0.79, respectively). We also found that in patients with AML and MDS, age was associated with a suboptimal BMA (OR 0.63; 95% CI 0.49-0.81 and OR 0.72; 95% CI 0.53-0.97), whereas high levels of hemoglobin (OR 2.56; 95% CI 2.07-2.98 and OR 1.16; 95% CI 1.02-1.35), and platelets (OR 1.27; 95% CI 1.07-1.44 and OR 1.34; 95% CI 1.11-1.49) were associated with an optimal BMA.Conclusions We found that in AML and MDS, factors such as advanced age, low levels of hemoglobin, platelets, and white blood cells are associated with the quality of the BMA, thereby impacting its diagnostic accuracy.
Introduction Ocular graft-versus-host disease (oGVHD) is a frequent complication after allogeneic hematopoietic stem cell transplantation (alloHCT), with dry eye symptoms affecting up to 90% of patients, often beginning within 3 months post-transplant and persisting indefinitely. Current treatments target established disease, and evidence for prophylactic approaches is limited. This study evaluated the safety, tolerability, and early efficacy of topical tacrolimus (Tac) versus cyclosporine (Cys) for oGVHD prevention in the outpatient alloHCT setting. Objective Primary endpoint: safety/tolerability. Secondary endpoints: oGVHD prevention, ocular symptoms, quality of life (QoL), and graft-versus-relapse-free survival (GFRS). Methods Non-blinded phase I–II trial (NCT06348602) including adults (>18 y) undergoing outpatient peripheral blood alloHCT from matched sibling or haploidentical donors. Exclusion criteria: active ocular infection or intolerance to Tac/Cys. After engraftment, patients were randomized to Tac 0.03% ointment or Cys 0.1% solution, twice daily in both eyes for 12 months. Artificial tears were allowed. Systemic GVHD prophylaxis included post-transplant cyclophosphamide, a calcineurin inhibitor, and mycophenolate. Ophthalmologic exams were performed every 3 months NIH/ICCGVHD criteria. Statistical analysis used IBM SPSS v27, with p < 0.05 considered significant. Results Twenty-two patients were enrolled (54.2% Cys, 45.8% Tac); median age 35 y; 70% male. Haploidentical donors 75%; reduced-intensity conditioning 50%. Median CD34⁺ dose 8.94 × 10⁶/kg; neutrophil and platelet recovery at 13 and 12.9 days. Acute GVHD occurred in 62.5% (grade 3–4 in 20.8%), chronic in 37.5% (moderate-severe 16.7%). Relapse 33.3%, mortality 20.8%. Median event-free survival 66.4 weeks; GFRS 6.2 months. Confirmed oGVHD occurred in 2 patients (1 per group) and probable in 2 Cys All were mild (median onset 18.3 weeks). Dry eye symptoms were more frequent in Cys (61.5% vs 18.2%), with Schirmer <5 mm only in Cys 33.3% vs 0%. Both treatments were well tolerated: burning 70% Cys vs 55% Tac; pruritus 15% vs 45%; blurred vision 30.7% vs 36.4%. No discontinuations occurred. Adherence was high (Tac 81.8%, Cys 76.9%). NEI VFQ-25 scores were similar: baseline 79.7 ± 16.0 vs 74.9 ± 23.9; engraftment 78.3 ± 15.5 vs 74.3 ± 14.5; 3 months 79.3 ± 14.5 vs 79.0 ± 7.5; p > 0.05. QoL remained stable at 12 months, Wilcoxon p = 0.93 Cys; p = 0.50 Tac Conclusions Both topical Tac and Cys were safe, well tolerated, and associated with high adherence in outpatient alloHCT. Tac showed a trend toward fewer dry eye symptoms and lower ocular toxicity, though not statistically significant. Ocular QoL remained preserved in both groups, supporting the feasibility of topical immunomodulator prophylaxis for oGVHD. Longer follow-up is warranted to confirm sustained benefit.
Introduction Total marrow and lymphoid (TMLI) irradiation may keep the benefits of total body radiation but reduce toxicity to healthy tissues in patients with high risk acute lymphoblastic leukemia (ALL). Thus, our objective was to study the feasibility and safety of TMLI in the context of outpatient allogeneic hematopoietic cell transplantation (HCT). Objectives Primary outcome was safety defined adverse events (CTCAE, v5.0) & early mortality. Secondary endpoints were D60 measurable residual disease, non-relapse mortality (NRM), relapse free survival (RFS), cumulative incidence of relapse (CIR) & overall survival (OS). Methods This was a phase I/II trial that included patients 16-45 years with relapsed or refractory ALL (NCT06209190). The conditioning was cyclophosphamide (Cy) 350 mg/m² and fludarabine 25 mg/m² (-6 to -4) followed by TMLI; 12 Gy in 6 fractions of 2 Gy BID (-3 to -1) using tomotherapy. For patients with prior central nervous system (CNS) infiltration, a cranial 6 Gy boost was administered. Peripheral blood cells were used. Graft-versus-host disease (GVHD) prophylaxis was post-transplant cyclophosphamide, calcineurin inhibitor and mycophenolate. All procedures were planned entirely on an outpatient basis with hospitalization only if clinically necessary. Results Fourteen patients with B-ALL were enrolled, with median age 20 years (16-41), 57% male, with 3 lines of prior therapy (2-4). Thirteen (93%) MRD negative before HCT. All TMLI sessions were performed successfully on an outpatient basis, 87% of sessions performed on schedule, with minor delays. Median CD34+ dose was 10 × 10⁶/kg (5.2–10.1). Recipients underwent haploidentical HCT (71%); 57% remained entirely as outpatients (n=8), 42.9% required hospitalization: 21.4% (n=3) for social support, 7.1% (n=1) due to oral intolerance, and 14.3% (n=2) for febrile neutropenia. G2-3 AE included hemorrhagic cystitis (n=3), without cases of severe mucositis or organ damage. G1-2 AE in >10% were all gastrointestinal. Cytomegalovirus infection occurred in n=5. Median neutrophil and platelet engraftment were 14 days (11-17) and 14 days (11-17) respectively. No early death was recorded ≤60 days post-HCT. Acute GVHD was 57.1% (n=8), including G1 in n=1, G2 in n=5, and G3 in n=2. Chronic GVHD was 35.7%, moderate in n=1, severe in n=4. One patient transplanted with active disease had primary failure and died after relapse and complications of a second transplant. At evaluation on D+60 100% were MRD-negative. Two patients relapsed (14%), one bone marrow, the other isolated CNS. 1-year OS, RFS, CIR and NRM rates were 81.3%, 80%, 24% and 7.1% respectively. The median follow-up was 11 months (2-17). Conclusion TMLI-based conditioning in adolescents and young adults with ALL was feasible and safe, allowing for a full-outpatient conduct in 57% of patients, supporting further studies assessing its efficacy and the safety of larger doses.
Introduction Hematopoietic cell transplantation (HCT) is a complex process that impairs nutritional status, resulting in a decrease in body fat and lean mass due to conditioning regimen, prior line of therapy, immunosuppressive therapy, and metabolic alterations that impact appetite and food intake.Bioelectrical impedance (BI) provides objective data on body composition, and instruments such as the Patient-Generated Subjective Global Assessment (PG-SGA) can identify patients with nutritional risk.In our institution, the process of HCT is ambulatory. The patients have an intervention in the nutritional area with nutritional indications, but it is important identify patients with nutritional risk and do and carry out a more intense intervention. Objectives Primary objective: cumulative incidence of hospitalization (CIH) according to pre-transplant nutritional status as defined by the PG-SGA.Secondary objectives: cumulative Incidence of infection, non-relapse mortality (NRM), overall survival (OS), and relapse-free survival (RFS). Methods A prospective study was conducted at our outpatient HCT center, aged 18–75 years. Before conditioning, all participants underwent a nutritional evaluation by a licensed nutritionist, which included BI analysis and the PG-SGA. The evaluation was repeated at day +30 (±10) post-transplant. Results A total of 85 patients have been enrolled, 38% (n = 32) females and 62% (n=53) males. The median age was 45 (IQR 18-72) years. Most patients had a 0-1 ECOG (98%, n = 83), an HCT-CI of 0 (76%, n = 65), and were enrolled after a median of 2 (IQR 1-3) lines of therapy. The most common transplant indication was acute myeloid leukemia (27%, n = 23), followed by acute lymphoid leukemia (26%, n = 22) and multiple myeloma in third (24%, n=25%). HCT type: autologous (42%, n = 36), haploidentical (36%, n = 31).At baseline, 66% were well-nourished and 34% nutritional risk. The 100-day cumulative incidence of hospitalization was significantly higher in malnourished patients compared to well-nourished ones (56% vs. 36%; p = 0.011). In Fine-Gray regression, baseline malnutrition was associated with an increased risk of hospitalization (HR 2.24; 95% CI, 1.22–4.09; p = 0.009).The 100-day cumulative incidence of infection was higher among nutritional risk patients (61% vs 34%, p = 0.03, HR 2.01, 95%CI 1.06-3.81). Significant differences were also observed in 100-day NRM (13% vs. 0%, p = 0.02).One-year OS and RFS were superior in well-nourished (96% vs 70%, p = 0.024, and 91.5% vs 62%, p = 0.006, respectively).At day +30, hospitalized patients exhibited greater fat and muscle mass loss; 53% of well-nourished patients were reclassified as being at nutritional risk. Conclusion Pre-transplant nutritional status, as determined by PG-SGA, showed a significant association with the need for hospitalization and infections in patients undergoing outpatient HCT.
The therapeutic approach to smoldering multiple myeloma (SMM) is shifting from observation to early treatment, particularly in patients at high-risk of progression. Patients with SMM may experience factors that can negatively affect health-related quality of life (HRQoL). In addition, early therapeutic interventions may themselves impact HRQoL. Given these considerations, we aim to evaluate the assessment of HRQoL in clinical trials involving patients with SMM receiving treatment. We searched MEDLINE, Scopus, Web of Science, EMBASE, PubMED, and Cochrane Central Register of Controlled Trials for clinical trials assessing pharmacological interventions in patients with SMM from inception to July 2025. Study eligibility, data extraction and risk of bias assessment were conducted independently and in duplicate. The quality of evidence was assessed with the ROB-2 (risk of bias) assessment for randomized clinical trials. This study is registered on PROSPERO (CRD420251112091). The lack of consistent HRQoL assessment across trials stands out as a critical finding. Four randomised clinical trials evaluated HRQoL, although only 2 provided enough detail. Both the AQUILA and ECOG-ACRIN trials reported improvements in HRQoL; however, these benefits appeared to be treatment specific. However, despite these findings, most studies did not include HRQoL outcomes, highlighting a significant gap. This omission represents not only a methodological limitation but also as an ethical issue, given the importance of understanding treatment impact on patients' lives'. As therapeutic strategies continue to expand, the systematic incorporation of HRQoL outcomes is essential to support balanced and patient-centered decision-making.
Allogeneic hematopoietic stem cell transplantation (HSCT) is an effective treatment for B-cell acute lymphoblastic leukemia (B-cell ALL). An important mechanism for the prevention of relapse is the graft-versus-leukemia (GVL) effect, potentially at the expense of the accompanying graft-versus-host disease (GVHD), which has varying morbidity and mortality depending on its timing and grade. The association between the development of acute GVHD and chronic GVHD and a reduced incidence of relapse in B-cell ALL has been documented extensively, although conflicting results have been described in certain settings, including haploidentical HSCT followed by post-transplant cyclophosphamide (PTCy). We retrospectively examined the transplant-related outcomes of B-cell ALL adult patients undergoing HLA-matched related donor HSCT or haploidentical HSCT plus PTCy in a single center, and its association with acute and chronic GVHD. We included 43 patients with a median age of 22 years, of whom 65.1% were male. The graft source was peripheral blood stem cells in all cases. The donor source was HLA-matched related in 30.2% of cases, and haploidentical in 69.8%. Moreover, the conditioning regimen was myeloablative in 30.2% and reduced-intensity in 69.8%. Most patients underwent HSCT in first or second complete remission (44.2% each). Measurable residual disease was positive in 11.6% of patients prior to HSCT. The 2-year cumulative incidence of relapse, non-relapse mortality and overall survival (OS) were 33.3%, 7.4% and 65.5%, respectively. In a multivariable Cox-regression analysis, we found no association between acute GVHD (HR 0.94, 95% CI 0.28-3.15, p = 0.92), mild chronic GVHD (HR 0.56, 95% CI 0.06-5.23, p = 0.61) or moderate-severe chronic GVHD (HR 0.49, 95% CI 0.04-4.86, p = 0.54) and the occurrence of relapse. Also, there was no association between acute or chronic GVHD of any grade and OS. A positive MRD status showed an association with the incidence of relapse (HR 2.32, 95% CI, 0.55-9.77, p = 0.24) and OS (HR 2.17, 95% CI, 0.55-8.51, p = 0.26). In conclusion, acute and chronic GVHD were not related to differences in relapse or survival after allogeneic HSCT followed by PTCy-based GVHD prophylaxis. This underscores the importance of reappraising the role of GVHD and GVL in evolving transplant settings.
Introduction Access to hematopoietic cell transplantation (HCT) in Latin America remains centralized, limited by scarce regional centers and socioeconomic barriers that restrict access to curative therapies. Ambulatory HCT programs have emerged as safe, cost-efficient alternatives, but patients from distant areas may face additional challenges related to travel and follow-up. This study evaluated early outcomes and potential disparities among patients treated at our ambulatory HCT center in Mexico, which serves both national and international populations. Methods We conducted a retrospective cohort study of all pediatric and adult HCTs performed between January 2020 and December 2024 at a single FACT-accredited outpatient program in Monterrey, Mexico. Variables included age, sex, diagnosis, HCT type, time from diagnosis to HCT (“time to HCT”), time from first visit to transplant (“door-to-HCT”), and follow-up duration. Patients were categorized as local (Monterrey metropolitan area) or non-local (outside metro area, national, or international). Early outcomes included non-relapse mortality (NRM) and graft failure (GF) using competing-risk models; overall survival (OS) was measured from day 0 of HCT to last contact. Results A total of 489 HCTs in 476 patients were analyzed. Median age was 30 years (range 1–79), and 60.3% were male. Diagnoses included acute leukemia (43.4%), multiple myeloma (18.2%), lymphoma (18.0%), and bone marrow failure (10.2%). HCT types were haploidentical (42.5%), autologous (41.7%), and matched allogeneic (15.7%). Patients originated from 30 of 32 Mexican states and nine countries, mainly Guatemala (2.5%), Paraguay (1.4%), and Honduras (1%). Non-local patients (68.3%) were older (32 vs. 22 years), had shorter door-to-HCT intervals (22 vs. 38 days), and shorter follow-up (4 vs. 8 months), all p<0.01, with no differences in diagnosis, HCT type, or time to HCT. The 100-day NRM was 1.0% (autologous), 5.3% (matched), and 14.2% (haploidentical); GF was 0%, 2.7%, and 6.9%, respectively. One-year OS was 97%, 70.8%, and 68.7% (p=0.3), with no differences by geographic origin. Conclusions Our ambulatory HCT center safely delivers transplantation to a geographically diverse population. Despite longer travel distances, non-local and international patients achieved early outcomes comparable to local patients, supporting the feasibility and equity potential of outpatient HCT in resource-limited settings.
Multiple myeloma (MM) is a malignant hematologic disease marked by the abnormal growth of plasma cells, resulting in serious complications such as bone fractures, hypercalcemia, renal dysfunction, and anemia. It represents the second most frequent hematologic malignancy and is usually diagnosed at an advanced stage in Latin America. A multinational survey included 16 experts from 11 Latin American countries to share their experiences in myeloma healthcare, discuss existing gaps, and propose solutions. The public healthcare system struggles with slow diagnosis and treatment access, particularly in rural areas. The private system faces challenges related to insurance coverage and costs. A significant gap exists between the need for diagnostic tests and their availability. Access to new cancer treatments is hindered by inadequate public policies, high drug costs, and inconsistent approval processes. A comprehensive approach for improving MM management in Latin America is crucial, emphasizing training, access, stakeholder engagement, data enhancement, and financial resources.
Objective To evaluate immunoglobulin G antibody responses against Severe Acute Respiratory Syndrome Coronavirus 2 in multiple myeloma patients in the northeastern region of Mexico following different COVID-19 vaccination regimens. Methods This retrospective study included 33 multiple myeloma patients, and 28 healthy controls vaccinated with mRNA (BNT162b2, mRNA-1273) or adenovector (AZD1222) vaccines. Anti-spike receptor-binding domain (RBD) and anti-nucleocapsid immunoglobulin G levels were measured. Laboratory parameters, including monocyte and lymphocyte counts and serum-free light chains, were analyzed. Statistical comparisons used ANOVA, Kruskal-Wallis, and logistic regression. Results No significant differences in anti-spike receptor-binding domain immunoglobulin G levels were observed between multiple myeloma patients and healthy controls after one vaccine dose. Multiple myeloma patients who received ≥3 doses showed higher anti-spike receptor-binding domain immunoglobulin G levels compared to single-dose recipients. Anti-nucleocapsid antibodies (indicative of prior subclinical infection) were detected in 51.5% of multiple myeloma patients and correlated with elevated anti-spike receptor-binding domain levels. Patients in the third anti-spike immunoglobulin G quartile (10,427.9–23,954.0 AU/mL) had higher monocyte counts compared with patients in the lower quartiles. No correlations were found between anti-spike immunoglobulin G and lymphocyte counts, serum proteins, or gamma globulins. Abnormal kappa/lambda light chain ratios did not impair antibody responses in nucleocapsid-positive patients. Conclusion Multiple myeloma patients achieved comparable immunoglobulin G responses to healthy controls after one vaccine dose, with enhanced responses following ≥3 doses. Subclinical infections may augment humoral immunity. Elevated monocyte counts are indicative of innate immune activation following the administration of the vaccine. These findings support prioritizing booster doses for multiple myeloma patients despite immunosuppressive therapies.
BACKGROUND:Febrile neutropenia, a frequent complication in patients undergoing autologous stem cell transplantation, increases morbidity and hospitalization costs. OBJECTIVE:The aim of this study was to compare the efficacy of two formulations of pegylated filgrastim with filgrastim in patients who received autologous stem cell transplantation as outpatients for lymphoma or myeloma. METHODS:Thirty patients were randomized to receive a single 6 mg dose of either reference or biosimilar pegfilgrastim on Day +1. A retrospective Control Group of fifty-three patients who received filgrastim was included. MAIN RESULTS:The median times to neutrophil engraftment were 10, 9 and 11 days for the innovator pegfilgrastim, biosimilar pegfilgrastim (p-value = 0.14), and filgrastim (p-value = 0.0001), respectively. The median times to platelet engraftment were 10 days for both of the pegfilgrastim groups and 12 days for the filgrastim group (p-value = 0.0001). Febrile neutropenia incidence was lower in the pegfilgrastim group (6.7%) than in the filgrastim group (24.5%; p-value = 0.04). Cost analysis showed higher costs for the innovator pegfilgrastim, with filgrastim having the lowest cost of the three formulations. CONCLUSION:The innovator and the biosimilar pegfilgrastim demonstrated similar efficacy in engraftment speed and febrile neutropenia incidence. Pegfilgrastim was associated with faster engraftment, a lower incidence of platelet transfusion, and a lower incidence of febrile neutropenia.