
INTRODUCTION:The three most common myeloid/lymphoid neoplasms (MLN) with eosinophilia and tyrosine kinase gene fusions have rearrangements of the genes coding the alpha and beta subgroups of platelet-derived growth factor receptors (PDGFRA, PDGFRB) and fibroblast growth factor receptor one (FGFR1). The most common partner gene of PDGFRA rearrangement is FIP1L1. However, a few notable exceptions to this have been reported. CASE PRESENTATION:In our report, we present the case of a patient with high symptom burden and peripheral blood basophilia who initially went undiagnosed. Then, an unusual PRKG2::PDGFRA translocation with next-generation sequencing panel was detected. Treatment with imatinib led to complete remission. We discuss the clinical presentation, review the literature, and comment on its pathogenesis. CONCLUSION:In patients with myeloproliferative features, the absence of peripheral blood eosinophilia does not rule out MLN with tyrosine kinase gene fusions. Next-generation sequencing panels that detect RNA fusions may help uncover cryptic or rare fusions. MLN with PDGFRA rearrangement responds well to imatinib.
OBJECTIVE:This study evaluated the predictive value of plasma sPECAM-1, t-PAIC, and sCLEC-2 (individually and in combination) for early severe bleeding events in patients with acute promyelocytic leukemia (APL). METHODS:A retrospective review of 319 APL patients was conducted. Patients were divided into Occurrence and Non‑occurrence groups based on severe bleeding events. Plasma markers were measured. Logistic regression, ROC, calibration, decision curve analyses, and internal validation were performed. RESULTS:Plasma sPECAM‑1, sCLEC‑2, and t‑PAIC were significantly elevated in the Occurrence group. Elevated levels of these markers, along with reduced levels of FIB were independently associated with early severe bleeding events in patients with APL. AUCs for sPECAM-1, sCLEC-2, and t-PAIC were 0.759, 0.855, and 0.820, respectively; the three‑marker combination achieved an AUC of 0.929, outperforming any single marker and the traditional model (DIC score, FIB, D-D, and WBC) (AUC = 0.804). Adding the new biomarkers to the traditional model further increased the AUC to 0.959 (P < 0.001). Internal validation yielded a corrected AUC of 0.916, with good calibration (P = 0.069). The combined model provided net clinical benefit across threshold probabilities of 2-98%. CONCLUSION:Plasma sPECAM‑1, t‑PAIC, and sCLEC‑2 are elevated in APL patients with early severe bleeding. Their combined detection demonstrates high predictive efficacy for early severe bleeding in APL. However, the generalizability of this model requires further validation through large‑scale, multicenter, prospective studies.
Introduction: Acute promyelocytic leukemia (APL), a subtype of acute myeloid leukemia, typically presents with severe bleeding. However, the occurrence of APL with isolated myeloid sarcoma (MS) as the sole initial presentation is extremely rare, lacking typical clinical and laboratory features, which complicates diagnosis and management. Case Presentation: A 45-year-old male was admitted for 2-month lower back pain. Spinal MRI showed T10 vertebral signal changes; histopathology confirmed the vertebral mass was myeloid origin, with PML::RARα fusion detected via PCR. Initial blood counts, coagulation tests, and bone marrow (BM) examinations were normal. However, 21 days postoperatively, the patient’s platelet count and fibrinogen level declined. A subsequent BM examination revealed abundant abnormal promyelocytes, and FISH confirmed the presence of the PML::RARα fusion gene, diagnosing APL with MS and central nervous system (CNS) involvement. He achieved complete remission with all-trans retinoic acid-based chemotherapy. Conclusion: This is a rare case of isolated thoracic vertebra promyelocytic sarcoma as the initial symptom, which later progressed to APL with MS and CNS involvement. Additionally, we performed a comprehensive literature review of 13 other cases of APL with spinal MS at the time of diagnosis. In aggregate, APL presenting with spinal MS as the sole initial manifestation is extremely rare.
INTRODUCTION:Treatment-free remission (TFR) is an established goal for patients with chronic myeloid leukemia in chronic phase (CML-CP) who achieve sustained deep molecular response (DMR) on tyrosine kinase inhibitor (TKI) therapy. However, the relationship between pre-discontinuation dose intensity and TFR outcome remains poorly characterized. METHODS:We retrospectively analyzed 146 patients with CML-CP who attempted TFR at Samsung Medical Center between 2003 and 2025. Dose intensity (DI) was defined as the time-weighted average ratio of actual to standard TKI dose during the 2-year period preceding discontinuation. The association between DI and TFR failure was modeled using Cox regression with restricted cubic splines (RCS) in the DI subcohort (n=126), using a DI of 1.0 as the reference. RESULTS:In the full cohort, at median follow-up of 22.3 months, 50 patients (34.2%) experienced molecular relapse. TFR probability was 79.5% at 6 months and 61.9% at 48 months. In the DI subcohort, an unadjusted RCS model suggested a nonlinear, inverted U-shaped pattern, but the overall DI effect was not significant (likelihood ratio P = .101; P for nonlinearity = .045). In exploratory post-hoc groups, intermediate DI showed the highest TFR failure rate (49.0% vs. 25.0% and 28.6%; P = .035). The pattern did not reach significance after adjustment for TKI generation (P for nonlinearity = .074). CONCLUSION:These exploratory findings suggest that the biology underlying DMR maintenance, rather than the absolute pre-discontinuation dose, may influence TFR outcome. Given the modest sample size and post-hoc design, this hypothesis-generating observation requires confirmation in independent cohorts.
BACKGROUND:Multiple myeloma (MM) remains an incurable hematological malignancy with heterogeneous outcomes, necessitating the identification of novel prognostic markers for improved patient stratification and treatment. METHODS:This study investigated the prognostic significance of disulfidptosis-related genes (DRGs) in MM, analyzing 1,272 samples from TCGA_MMRF and GSE4581 datasets. To explore the biological functions of key DRGs, si-RNA was used to conduct in vitro experimental verification. RESULTS:Through comprehensive bioinformatic analyses, we identified four genes (FLNA, GYS1, LRPPRC, and MYH10) significantly associated with MM prognosis. These DRGs linked the signature to disulfidptosis biology in MM. A novel four-gene signature effectively stratified patients into high-risk and low-risk groups with distinct survival outcomes, remained an independent prognostic factor, and varied significantly across disease stages. High-risk scores were associated with pathways involving mRNA processing, DNA replication, and cell cycle progression, as well as immune shifts in neutrophil and plasma-cell fractions. Drug-sensitivity modeling showed distinct IC50 patterns between risk groups, with LRPPRC and GYS1 negatively correlated with sensitivity to selected agents. In vitro, LRPPRC knockdown in RPMI 8226 and U266 reduced viability and colony formation while increasing apoptosis, supporting the biological relevance of the signature. CONCLUSION:This novel DRGs signature not only provides a valuable tool for patient stratification but also offers insights into the biological processes driving MM progression and potential therapeutic targets.
Introduction The Revised International Prognostic Scoring System (IPSS-R) has long been the standard prognostic tool in the management of myelodysplastic syndromes (MDS). Recently, a new clinical-molecular prognostic model, the IPSS-M, was introduced. This model integrates MDS-related gene mutations and offers improved accuracy and precision in predicting patient outcomes and survival. In this study, we compare the prognostic value and predictive capacity of these prognostic scores across different treatment groups. Methods IPSS, IPSS-R, and IPSS-M scores were calculated at diagnosis for all eligible MDS patients in this cohort. Sankey diagrams were generated to visually compare the risk stratification across the three scoring systems. Results A total of 394 patients were included in this retrospective analysis. During the course of the disease, 59.1% required treatment, with 38.1% (150 patients) receiving erythropoiesis-stimulating agents (ESA) and 19.0% (75 patients) treated with hypomethylating agents (HMA). The IPSS-M was calculated for 281 patients and reclassified 41.9% of cases: 20.3% (n=57) were upstaged and 21.7% (n=61) were downstaged. All three scoring systems demonstrated significant stratification of overall survival (OS) in the global cohort (p<0.001 for IPSS, IPSS-R, and IPSS-M). Notably, IPSS failed to predict OS in patients treated with azacitidine, and none of the scores successfully predicted OS in patients treated with ESA. Conclusion The IPSS-M effectively reclassified 41.9% of patients, upstaging 20.2% and downstaging 21.7%. However, our analysis found that none of the prognostic scores effectively predicted outcomes for ESA-treated patients. Further studies are needed to assess whether the predictive value of these scoring systems is influenced by the specific treatment modalities used.
INTRODUCTION:Patients with homozygous β-thalassemia show continuously improved survival, which has contributed to the development of kidney complications, predominantly expressed as increased albuminuria. In particular, patients with transfusion-dependent thalassemia frequently develop chronic kidney complications related to chronic anemia, iron overload, and exposure to iron chelation therapy. Although sodium-glucose co-transporter 2 inhibitors have demonstrated renoprotective effects in patients with chronic kidney disease (CKD) from other causes, their efficacy in patients with homozygous β-thalassemia and established CKD remains unknown. METHODS:In this prospective, open-label study, we investigated the impact of dapagliflozin (10 mg/day), in patients with homozygous β-thalassemia and an albumin-to-creatinine ratio (ACR) >30 mg/g. Study end-points included the effect on ACR and kidney function at 6 months after treatment initiation. Furthermore, we examined the effect on systolic blood pressure (SBP) and diastolic blood pressure, and on urine monocyte chemoattractant protein-1 (MCP-1) and matrix metalloproteinase-9 (MMP-9) levels. RESULTS:Twelve patients with ACR >30 mg/g received dapagliflozin, and 18 patients with normal ACR were followed up for 6 months. Twenty-three healthy adult individuals age- and sex-matched to the β-thalassemia group were used as normal controls. Patients with ACR >30 mg/g showed a significant reduction of ACR and SBP at 6 months (p = 0.034, p = 0.025, respectively), while kidney function remained stable. Patients with homozygous β-thalassemia showed increased baseline urine MCP-1 levels, while MMP-9 showed no differences versus normal controls. Those who received dapagliflozin exhibited a significant decrease in urine MCP-1 (p = 0.022), while MMP-9 remained stable. There were no significant adverse events. CONCLUSION:Dapagliflozin at a standard dose of 10 mg daily is safe and effectively reduces albuminuria and urinary MCP-1 in patients with homozygous β-thalassemia and established CKD, suggesting potential kidney anti-inflammatory effects. Further studies with larger cohorts and longer follow-up are needed to determine its impact on CKD progression and long-term outcomes.
INTRODUCTION:Second-generation FLT3 inhibitors have demonstrated clinical efficacy in FLT3-mutated acute myeloid leukemia (AML), although results across randomized trials remain inconsistent. This meta-analysis aimed to evaluate the efficacy of second-generation FLT3 inhibitors in patients with FLT3-mutated AML. METHODS:We systematically searched databases including PubMed, Web of Science, and the Cochrane Library (from inception to August 2025) to identify randomized controlled trials (RCTs) evaluating second-generation FLT3 inhibitors for treating FLT3-mutated AML. The primary endpoints were overall survival (OS) and progression-free survival (PFS). A random-effects model was used to calculate pooled hazard ratios (HRs) and their 95% confidence intervals (95% CIs). The study protocol was registered in PROSPERO (CRD420251132477). RESULTS:This meta-analysis included eight RCTs involving 2,231 patients. The second-generation FLT3 inhibitors evaluated were gilteritinib and quizartinib, and control treatments included placebo or active non-FLT3-inhibitor regimens. Patients treated with second-generation FLT3 inhibitors exhibited significant improvements in both OS (HR: 0.72, 95% CI: 0.62-0.84; p < 0.001) and PFS (HR: 0.73, 95% CI: 0.63-0.86; p < 0.001). Subgroup analysis revealed that gilteritinib showed improvements in both OS (HR: 0.71, p = 0.001) and PFS (HR: 0.71, p < 0.001), and quizartinib significantly improved OS (HR: 0.72, p = 0.013) with no significant difference in PFS (HR: 0.75, p = 0.142). Among patients with newly diagnosed AML, second-generation FLT3 inhibitors did not significantly improve PFS (HR: 0.73, p = 0.127) but significantly improved OS (HR: 0.80, p = 0.037). Among patients with relapsed/refractory AML, significant benefits were observed in both OS (HR: 0.67, p < 0.001) and PFS (HR: 0.74, p = 0.04). Benefits were observed across both placebo-controlled and active-comparator trials. CONCLUSION:Second-generation FLT3 inhibitors significantly improve survival outcomes in FLT3-mutated AML, particularly for gilteritinib and in relapsed/refractory disease. Further studies are needed to clarify mutation subtype-specific and dose-specific effects.
Introduction: Acute myeloid leukemia (AML) post myeloproliferative neoplasm (MPN) have very poor prognosis and are often excluded from most clinical trials. Methods: We retrospectively collected data from 166 patients, including 156 with available treatment data and 83 with NGS data treated in France and USA. Results: 2022 ELN risk categories were favorable, intermediate and adverse in 3 (2%), 17 (13%), and 110 (85%), respectively. Overall response rate was 57%, 20%, and 25% in patients treated by intensive chemotherapy (IC), hypomethylating agents (HMAs) and BSC (including low-intensity treatments as hydroxyurea and low-dose cytarabine), respectively. A total of 31 (22%) patients underwent allogeneic stem cell transplantation (ASCT). Median overall survival (OS) was 7.2 months without significant difference between IC and HMA (9.5 and 8.6 months, respectively). OS was significantly improved in patients allografted (6.7 vs. 1.3 months, respectively, p < 0.001). Even though 2017 and 2022 ELN risk categories were not prognostic for OS, we observed a prognostic impact on OS of Lindsley’s classifier (p = 0.014). In multivariate analysis, JAK2 mutation was associated with worse OS (p = 0.037), whereas SRSF2 showed a trend toward adverse prognosis (p = 0.057). Among functional groups, only spliceosome mutations predicted poor prognosis (p = 0.015). Conclusion: We confirmed poor prognosis of AML post-MPN. Classical prognostic classification was not validated in our cohort. We observed poor outcome using IC or HMA encouraging us to propose new clinical trials in this specific subgroup. Only ASCT was able to improve prognosis.
INTRODUCTION:This study aimed to characterize multiple myeloma (MM) patients at the first relapse and examine treatment outcomes. METHODS:This registry-based retrospective study examined relapsed MM patients in two Finnish Wellbeing Services Counties (Helsinki and Uusimaa, and Southwest Finland) from 2013 to 2022. Relapse was identified per current guidelines using serum paraprotein, plasma immunoglobulins, and free light chain data. Data were collected from hospital data lakes, Social Insurance Institution and Digital and Population Services Agency. Patients were stratified by age (≤70 vs. >70), index year, and time to first relapse (≤2 vs. >2 years post-diagnosis). RESULTS:Of 1,221 patients treated for MM between 2012 and 2022, 286 met the study inclusion criteria. Median overall survival from the relapse was higher in the younger patients (32.4 months vs. 20.0 months). In the first half of follow-up, lenalidomide was the most common pharmacotherapy in both age groups. After 2018, CD38-based treatments and pomalidomide increased significantly for younger patients, remaining limited in the elderly. CONCLUSION:While new therapies have been adopted for younger MM patients, the treatment of older patients at first relapse has had little change. Given their poorer outcomes, there is a need to improve care for older MM patients.
INTRODUCTION:The efficacy of donor lymphocyte infusions (DLIs) among various myeloid malignancies (particularly genetic subtypes) and the optimal timing of DLI initiation remains unclear. METHODS:This was a retrospective study of 62 patients with myeloid malignancies treated with alloHSCT and DLI from years 2001 to 2022. DLI indications were therapeutic 55 (89%), preemptive 6 (10%), and prophylactic in 1 (2%) patients with complete remission (CR/CRi) in 20 patients; diagnoses included 9 (45%) acute myeloid leukemia, 6 (30%) myelodysplastic syndromes, and 3 (15%) chronic myelomonocytic leukemia among others. RESULTS:Among patients who received therapeutic DLI (n = 55), the best response was CR/CRi in 16 (29%) patients. At a median follow-up from the date of DLI of 75 (95% CI 35-121) months, there were 49 (79%) deaths with a median OS of 6 (95% CI 4-14) months, higher in patients in CR/CRi versus no CR/CRi (median 51 versus 4 months, p = 0.008). Presence of cytogenetic abnormalities such as complex karyotype, deletions in chromosome 5, 7, and 17p, and mutations in TP53, KRAS, NRAS, RUNX1, or JAK2 were associated with adverse outcomes. CONCLUSION:DLI is an effective treatment strategy for post-transplant relapse across myeloid malignancies, with specific genetic subtypes showing poorer outcomes and survival.
INTRODUCTION:Coronavirus disease 2019 (COVID-19) poses significant risks to immunocompromised individuals. Patients receiving maintenance therapy with anti-CD20 monoclonal antibodies for follicular lymphoma (FL) may be especially vulnerable because of prolonged B-cell depletion. However, data from Japan, particularly from the later stages of the pandemic, remain limited. This study aimed to investigate the incidence, severity, duration, mortality, and treatment discontinuation associated with COVID-19 in this population. METHODS:We retrospectively analyzed all consecutive 72 patients with FL or related indolent B-cell lymphomas, including transformed FL, who received anti-CD20 maintenance therapy (rituximab or obinutuzumab) at our institute between January 2020 and September 2023. All patients had an indolent lymphoma background. Data on clinical features, laboratory values, disease severity at initial diagnosis according to Ministry of Health, Labour and Welfare guidelines, hospitalization, outcomes, duration of polymerase chain reaction positivity (>21 days defined as prolonged), and maintenance therapy continuation were reviewed. Fisher's exact test was used for analysis. RESULTS:Among the 72 patients (median age: 69 years), 16 (22.2%) developed COVID-19. Of these, 5 had received rituximab and 11 obinutuzumab. The median lymphocyte count at infection was 498/μL; no patient had serum IgG <500 mg/dL. Eight infected patients developed oxygen-requiring disease at initial assessment (50.0% of infected patients; 11.1% overall). Prolonged infection occurred in 9 patients (56.3% of infected patients; 12.5% overall). All four deaths were COVID-19-related and occurred in obinutuzumab recipients (25.0% of infected patients; 5.6% overall). Lymphocyte count <200/μL was associated with mortality (p = 0.027), although this finding should be interpreted cautiously because of the small number of events. Maintenance therapy was discontinued in 5 of 8 patients infected during therapy. CONCLUSION:COVID-19 infection during anti-CD20 maintenance therapy led to severe illness and prolonged viral shedding in some cases. Severe lymphopenia may be a potential risk marker for mortality, but this finding should be interpreted cautiously because of the exploratory nature of the analysis.
BACKGROUND:Tagraxofusp, a first-in-class CD123-targeted therapy, is the only approved treatment for blastic plasmacytoid dendritic cell neoplasm (BPDCN). Shared care models enable coordination and care delivery between specialized cancer treatment centers and local institutions, allowing eligible patients to receive tagraxofusp. SUMMARY:This review provides guidance on best practices for effective shared care implementation for tagraxofusp in patients with BPDCN, including appropriate patient selection, adequate local institutional readiness, proactive collaboration and communication between institutions, integrated comanagement between institutions, and requisite staff, patient, and caregiver education. KEY MESSAGES:Shared care is particularly suitable for tagraxofusp treatment, as adverse events can be grade 3 or greater, but typically only occur in cycle 1, if they are to occur at all. Based on clinical experience, this article discusses the application of a shared care model for tagraxofusp treatment in BPDCN.
INTRODUCTION:The aim of this study was to analyze the clinical infusion effect of frozen-thawed-deglycerolized red blood cells (FTD-RBCs) and provide solutions for situations such as blood supply shortages. METHODS:In this retrospective study, 280 patients receiving FTD-RBCs and 280 patients receiving suspended red blood cells (SRBCs) in our hospital from December 2019 to June 2022 were included. We collected the course records and laboratory test results, including basic information, total amount of transfusion, as well as RBC count, hemoglobin concentration (HGB), and hematocrit (HCT) 24 h before and after the transfusion, and whether there were adverse blood transfusion reactions. Clinical efficacy analysis of FTD-RBCs was conducted by Mann-Whitney U test and Kruskal-Wallis H test. RESULTS:In the 280 patients of our study, RBC count, HGB, and HCT increased significantly after transfusion of FTD-RBCs (p < 0.05). For transfusion of one FTD-RBC unit, the median differences in RBC count, HGB, and HCT before and after transfusion were 0.310 × 1012/L, 9.735 g/L, and 2.800%, respectively. The increments of the three indicators in the FTD-RBC group were all lower than those in the SRBC group, but the differences were not statistically significant (p > 0.05). None of the patients in this study had adverse transfusion reactions after transfusion of FTD-RBCs. In addition, we found that the efficacy of FTD-RBCs transfusion was associated with sex, age, body weight, disease type, and blood type of the patient (p < 0.05). CONCLUSION:The infusion of FTD-RBCs for clinical patients is safe and effective, and it is an effective blood supply supplement in situations of blood shortage such as public health events.
INTRODUCTION:Chronic myelomonocytic leukemia (CMML) is a biologically heterogeneous myelodysplastic/myeloproliferative neoplasm with limited disease-modifying treatment options beyond hypomethylating agents (HMAs) and allogeneic hematopoietic stem cell transplantation. Venetoclax, a selective BCL-2 inhibitor, is increasingly used off-label in CMML, but its CMML-specific efficacy and safety remain incompletely defined. METHODS:We conducted a systematic review and meta-analysis in accordance with PRISMA 2020. Eligible studies included adult patients with CMML treated with venetoclax-based regimens and reporting extractable CMML-specific outcomes. PubMed, Embase, Cochrane CENTRAL, conference proceedings, and trial registries were searched through August 2025. Random-effects meta-analyses of proportions were performed for complete remission (CR), marrow complete remission (mCR), and overall response rate (ORR). RESULTS:Seventeen publications representing nine unique studies were included, comprising 145 venetoclax-treated CMML patients. Most regimens combined venetoclax with azacitidine, decitabine, or oral decitabine-cedazuridine, with heterogeneous dosing schedules and frequent CYP3A-guided dose modifications. Responses typically occurred early, within one to two treatment cycles, but durability was generally modest. The pooled CR rate was 19.1% (95% CI: 9.4-34.9; I2 = 55%), the pooled mCR rate was 36.4% (95% CI: 24.7-50.0; I2 = 21%), and the pooled ORR was 71.9% (95% CI: 56.5-83.4; I2 = 56%). Venetoclax-based therapy was associated with substantial myelosuppression, including frequent grade ≥3 neutropenia and thrombocytopenia, with clinically relevant infectious complications. Early mortality was low in studies reporting short-term outcomes. CONCLUSION:Venetoclax-based regimens demonstrate measurable but limited activity in CMML, with high overall response rates but low CR rates and modest durability. Prospective CMML-specific trials are needed to define optimal dosing, clarify comparative effectiveness, and identify patients most likely to benefit.
INTRODUCTION:Measurable residual disease (MRD) assessment in Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) commonly relies on reverse transcription polymerase chain reaction detection of BCR::ABL1 transcripts. However, discordance between BCR::ABL1 PCR and next-generation sequencing (NGS)-based MRD assays targeting immunoglobulin/T-cell receptor (IG/TR) rearrangements may occur, particularly in cases of multilineage Ph+ ALL, complicating treatment decisions such as tyrosine kinase inhibitor (TKI) discontinuation. CASE PRESENTATION:We report a 54-year-old female with Ph+ ALL who achieved durable remission with sustained NGS MRD negativity by IG/TR despite persistent low-level BCR::ABL1 transcript positivity. Following self-discontinuation of ponatinib, she experienced a rapid rise in BCR::ABL1 transcripts and loss of cytogenetic remission in myeloid cells, while remaining morphologically in remission and NGS MRD-negative. Reintroduction of ponatinib resulted in a prompt transcript decline. CONCLUSION:This case highlights the clinical significance of multilineage Ph+ ALL and its distinct biological background, thereby underscoring caution with TKI discontinuation despite deep NGS MRD negativity and may support continued TKI therapy in this setting.
INTRODUCTION:Newly diagnosed autoimmune hemolytic anemia (AIHA) develops in 1-3 individuals per 100,000 people per year. Warm autoimmune hemolytic anemia (WAIHA) is the most common type of AIHA, accounting for approximately 70-80% of cases. This report outlines a complex presentation of DAT-negative, anti-e-mimicking autoantibody mediated WAIHA following a transient viral infection in an adult male patient with no prior transfusions. CASE PRESENTATION:The 55-year-old male patient presented with several weeks of flu-like symptoms followed by jaundice, dyspnea, and progressive fatigue. Initial laboratory workup revealed hemolytic anemia, and subsequent serologic testing identified a warm autoantibody with anti-C specificity, but was ultimately consistent with an anti-e mediated WAIHA. CONCLUSION:This report highlights a complex case of a patient with an autoantibody mimicking anti-e associated WAIHA and the challenge of identifying suitable transfusion options.
INTRODUCTION:The prognosis and management of pregnancy-associated aplastic anemia (PAA) following allogeneic hematopoietic stem cell transplantation (allo-HSCT) remain uncertain. This retrospective study was conducted to describe our clinical experience and to explore the efficacy and safety of allo-HSCT in patients with PAA. METHODS:A retrospective observational study was evaluated the safety and feasibility of allo-HSCT in 18 patients with PAA who were treated between January 2013 and November 2023. RESULTS:Median neutrophil engraftment occurred at 11.5 days (range: 6-15), and platelet engraftment occurred at 11.5 days (range: 7-28). The cumulative incidence (CuI) of both neutrophil and platelet engraftment was 100%. The CuI rates were 22.96% (95% CI, 13.68-27.32) for grade II acute graft-versus-host disease (aGVHD) and 12.61% (95% CI, 9.32-29.28) for chronic GVHD (cGVHD). No cases of grade III-IV aGVHD, extensive cGVHD, or relapse were observed. The CuI of transplantation-related mortality was 19.87% (95% CI, 13.15-30.73). Among the 15 surviving patients, the median follow-up was 47.0 months (range: 7.6-122.2 months). The overall survival and relapse/rejection-free rates were both 80.14% (95% CI, 69.27-86.86). CONCLUSION:These findings suggest that allo-HSCT is a viable and effective treatment option for PAA. The donor types included 8 matched sibling donors, 1 matched unrelated donor, 3 mismatched unrelated donors, and 6 haploidentical donors.
Introduction: Lymphoma has recovery rates above 60%, but many survivors experience impaired quality-of-life (QoL) requiring survivorship care. This study evaluated the role of an integrative oncology (IO) clinic in managing lymphoma survivors. Methods: In this exploratory preference-based controlled trial, adults in remission after lymphoma treatment were allocated to two groups: those attending the IO survivorship clinic (intervention) and those declining (control). The intervention included complementary medicine, spiritual, and social support, delivered weekly for up to 6 months in addition to standard follow-up. The primary outcome was QoL improvement (EQ-5D-5L index). Secondary outcomes included symptom relief (MYCAW), cognitive function, and perceived control. Results: Twenty-nine patients were enrolled: 15 in the intervention and 14 in the control group. Over the first 3 months, a significant time × group interaction in EQ-5D-5L scores favored the intervention (p = 0.005), reflecting superior QoL trajectory. MYCAW concerns also improved significantly in the intervention group across 6 months (p = 0.005 and p = 0.03). At 3 months, FACT Cog-Oth scores were significantly higher in the intervention arm (p = 0.01), indicating better “other” cognitive functions (e.g., memory, clarity, confusion). To note, adherence to IO mainly decreased after 3 months. Conclusion: In this exploratory preference-based study, an IO survivorship clinic for lymphoma survivors was associated with improvements in QoL, especially for adherent patients. Given the preference-based design, these findings should be interpreted cautiously and viewed as hypothesis-generating rather than confirmatory. Further studies are warranted to evaluate long-term benefits and sustainability of this approach.