{"title":"The Synthetic Image Crisis in Science","authors":"Enrico M. Bucci, Angelo Parini","doi":"10.1002/ajh.27697","DOIUrl":"10.1002/ajh.27697","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 7","pages":"1114-1117"},"PeriodicalIF":10.1,"publicationDate":"2025-04-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.27697","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143880210","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Lindsay M. Olson, Mark Gurney, Marissa Li, Yuan Yao, Tucker Johnson, Horatiu Olteanu, Naseema Gangat
{"title":"Acute Megakaryoblastic Leukemia Masquerading as a Germ Cell Tumor","authors":"Lindsay M. Olson, Mark Gurney, Marissa Li, Yuan Yao, Tucker Johnson, Horatiu Olteanu, Naseema Gangat","doi":"10.1002/ajh.27698","DOIUrl":"https://doi.org/10.1002/ajh.27698","url":null,"abstract":"<h2> Conflicts of Interest</h2>\u0000<p>The authors declare no conflicts of interest.</p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"79 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-04-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143880227","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Rocky Mountain Spotted Fever, a Lymphoma Mimic on Peripheral Blood Smear","authors":"Patricia Lupu, Linnea Banker, Richard D. Hammer","doi":"10.1002/ajh.27695","DOIUrl":"https://doi.org/10.1002/ajh.27695","url":null,"abstract":"<h2> Conflicts of Interest</h2>\u0000<p>The authors declare no conflicts of interest.</p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"25 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-04-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143880209","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Tobias Dittrich, Timon Hansen, Christoph R. Kimmich, Kaya Veelken, Anna Jauch, Marc S. Raab, Carsten Müller-Tidow, Ute Hegenbart, Stefan O. Schönland
{"title":"Elotuzumab in Combination With Dose Reduced IMiDs and Dexamethasone for AL Amyloidosis Patients With Relapsed/Refractory Plasma Cell Dyscrasia and Advanced Organ Involvement","authors":"Tobias Dittrich, Timon Hansen, Christoph R. Kimmich, Kaya Veelken, Anna Jauch, Marc S. Raab, Carsten Müller-Tidow, Ute Hegenbart, Stefan O. Schönland","doi":"10.1002/ajh.27684","DOIUrl":"10.1002/ajh.27684","url":null,"abstract":"<p>Systemic light chain (AL) amyloidosis is a rare, life-threatening disorder characterized by toxic light chain deposition and multiorgan dysfunction [<span>1, 2</span>]. Standard treatment targets the clonal plasma cells, and daratumumab-based regimens have demonstrated rapid hematologic responses with significant organ recovery [<span>3</span>]. However, with the increasing use of daratumumab in early treatment regimens, there is a growing need for effective therapies for AL patients with double or triple refractory plasma cell disorders. Elotuzumab, a humanized IgG1 monoclonal antibody targeting SLAMF7 on plasma cells and natural killer cells, has been proposed as a promising option in case of daratumumab failure in multiple myeloma, particularly when used in combination with pomalidomide [<span>3</span>]. Although its combination with immunomodulatory drugs (IMiDs) appears promising for AL amyloidosis, data remain scarce, highlighting the need for further investigation to better define its role in patients with advanced organ involvement.</p><p>We retrospectively evaluated 33 AL patients with relapsed/refractory plasma cell disorder who were initiated with elotuzumab, dexamethasone, and an IMiD (pomalidomide in 25 patients) between January 2017 and October 2022. Table S1 displays the patient characteristics at initial diagnosis of the AL amyloidosis. All patients had the baseline visit prior to therapy start with elotuzumab and at least one follow-up visit at one of the two institutions, Heidelberg Amyloidosis Center or Hamburg Amyloidosis outpatient clinic (at Onkologicum HOPA). Elotuzumab was administered intravenously according to the manufacturer's prescribing guidelines along with standard premedication including antihistamines and dexamethasone. The initial dexamethasone dose was 20 mg, followed by 8 mg in subsequent treatments, with dose reductions considered in response to toxicity. The initial elotuzumab dose was 10 mg/kg total body weight administered weekly for the first 2 cycles, followed by 10 mg/kg total body weight every 2 weeks (combination with lenalidomide) or 20 mg/kg total body weight every 4 weeks (combination witch pomalidomide) until disease progression or unacceptable toxicities emerged. Additionally, patients orally received pomalidomide and lenalidomide on days 1–21 of each cycle, with median doses of 3 mg (range: 2–4 mg) and 7.5 mg (range: 2.5–15 mg), respectively. As of the data-cutoff date on August 4, 2024, a median of five elotuzumab cycles were administered (range: 2–46). Data acquisition, response evaluation, evaluation of tolerability and adverse events as well as statistical methods are detailed in the supplemental information.</p><p>At the start of elotuzumab (Table S2), the cohort exhibited significant organ dysfunction (27% on dialysis; 50% with NT-proBNP > 8500 ng/L) and extensive pretreatment (median of three prior lines, with 79% refractory to proteasome inhibitors, 55% to IMiDs, and 85% to daratum","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 6","pages":"1098-1101"},"PeriodicalIF":10.1,"publicationDate":"2025-04-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.27684","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143872818","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Joanna Kamaso, Rocío García-Serra, Marina Munné, María Rodríguez-Rivera, Carme Melero, Sílvia Ramos-Campoy, Marta Salido, Marta Lorenzo, Eva Gimeno, Joan Gibert, Peter Vandenberghe, Katrina Rack, Anna Puiggros, Barbara Dewaele, Blanca Espinet
{"title":"Integrating Optical Genome Mapping With TP53 FISH: A Synergistic Approach for Cytogenomic Analysis in Chronic Lymphocytic Leukemia","authors":"Joanna Kamaso, Rocío García-Serra, Marina Munné, María Rodríguez-Rivera, Carme Melero, Sílvia Ramos-Campoy, Marta Salido, Marta Lorenzo, Eva Gimeno, Joan Gibert, Peter Vandenberghe, Katrina Rack, Anna Puiggros, Barbara Dewaele, Blanca Espinet","doi":"10.1002/ajh.27690","DOIUrl":"10.1002/ajh.27690","url":null,"abstract":"<p>Fluorescence in situ hybridization (FISH) is the gold standard technique for cytogenetic assessment in chronic lymphocytic leukemia (CLL). In addition, chromosome banding analysis (CBA) is recommended as part of testing to detect complex karyotypes (CK, ≥ 3 abnormalities in the same cell clone), especially as those with a high-CK (≥ 5 abnormalities) have a known worst outcome [<span>1, 2</span>]. Optical genome mapping (OGM) has emerged as a high-resolution technique to detect genome-wide balanced and unbalanced abnormalities, overcoming some disadvantages associated with current cytogenomic methods [<span>3</span>]. This study aimed to compare the effectiveness of OGM against CBA and FISH techniques in detecting poor prognostic cytogenomic biomarkers in CLL within a cohort of 102 CLL patients from two European centers, thus assessing the potential of OGM as a future routine diagnostic test.</p><p>Patients were selected to represent all the risk categories of the FISH-based Döhner hierarchical model: isolated del(13q) (<i>n</i> = 19), normal FISH (<i>n</i> = 18), trisomy 12 (<i>n</i> = 17), del(11q)(<i>ATM</i>) (<i>n</i> = 28), and del(17p)(<i>TP53</i>) (<i>n</i> = 20) (Table S1). OGM experiments were performed following the manufacturer's protocols, analyzed using the rare variant analysis pipeline (GRCh38/hg38 as a reference) and results visualized with the Bionano Access software (v1.7.2) (Bionano Genomics, San Diego, CA, USA). The detected structural variants (SVs) and copy number alterations (CNAs; gains and losses) were filtered out in different steps by discarding artifacts and polymorphisms, and applying filter settings based on confidence scores and size for the remaining alterations. The filtering strategy was based on criteria used in chromosomal microarrays analyses [<span>4</span>], and aimed to identify translocations, clinically relevant abnormalities in CLL independent of the size, and other abnormalities ≥ 5 Mb. Additionally, non-CLL abnormalities, sized between 200Kb and 5 Mb, were exclusively retained if they were part of a chromoanagenesis event or associated with other retained SV. First, we evaluated OGM's ability to detect abnormalities identified by the FISH panel and CBA. Next, we analyzed genomic complexity in patients stratified by CK status to determine a potential threshold for defining complexity by OGM. Statistical analyses were performed using SPSS v.23 software (SPSS Inc., Chicago, IL, USA). <i>p</i> values < 0.05 were considered statistically significant. Further details on methodology are described in Supporting Information.</p><p>Concerning cytogenomic abnormalities included in Döhner's model, OGM identified 90% (112/125) of those previously detected by FISH. Additionally, OGM detected two small deletions [one del(11q) and one del(13q)] undetectable by the standard FISH probes used in routine practice due to their small size (Figure 1A). Although OGM failed to identify 13 CNAs present in 6.5% to 17% of nu","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 7","pages":"1242-1245"},"PeriodicalIF":10.1,"publicationDate":"2025-04-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.27690","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143872817","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Claudia R. Morris, Dunia Hatabah, Rawan Korman, Scott Gillespie, Nitya Bakshi, Lou Ann Brown, Frank Harris, Deborah Leake, Chris A. Rees, Kirshma Khemani, Elliott P. Vichinsky, Alexus Locke, Bridget Wynn, Mark A. Griffiths, Hagar Wilkinson, Polly Kumari, Lisa Sudmeier, Sruti Shiva, Carlton D. Dampier
{"title":"Arginine Therapy for Pain in Sickle Cell Disease: A Phase-2 Randomized, Placebo-Controlled Trial","authors":"Claudia R. Morris, Dunia Hatabah, Rawan Korman, Scott Gillespie, Nitya Bakshi, Lou Ann Brown, Frank Harris, Deborah Leake, Chris A. Rees, Kirshma Khemani, Elliott P. Vichinsky, Alexus Locke, Bridget Wynn, Mark A. Griffiths, Hagar Wilkinson, Polly Kumari, Lisa Sudmeier, Sruti Shiva, Carlton D. Dampier","doi":"10.1002/ajh.27692","DOIUrl":"10.1002/ajh.27692","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <p>We present a prospective randomized, placebo-controlled trial of intravenous arginine in patients 3–21 years hospitalized with sickle cell disease vaso-occlusive pain episodes (SCD-VOE) at two tertiary-care children's hospitals. Participants were randomized into 1 of 3 arms: <i>Standard-dose</i> (SD; 100 mg/kg/dose) every 8 h, <i>Loading-dose</i> (200 mg/kg followed by SD), or <i>Placebo</i>. The primary outcome was total parenteral opioid use (TPO). Secondary outcomes included time-to-crisis-resolution, pain scores, patient-reported outcomes (PROs), arginine bioavailability, and biomarkers of oxidative stress/mitochondrial function. Of 1548 patients screened, 108 were randomized (36 per study-arm; mean 12.6 ± 3.8 years, 52% female, and 65% hemoglobin-SS). This study did not meet its primary endpoint. TPO, time-to-crisis-resolution, pain scores, and PROs at discharge were similar across arms. <i>Post hoc</i> sensitivity analyses of children 5–16 years old demonstrated nearly double TPO utilization in those receiving placebo versus arginine (<i>n</i> = 87, <i>p</i> = 0.056), achieving significance in patients with plasma arginine < 60 μM. Arginine was low at presentation in 79% of patients (mean 50 ± 28 μM), and increased with arginine therapy (<i>p</i> < 0.001). Arginine bioavailability at VOE presentation inversely correlated with time-to-crisis-resolution (<i>r</i> = −0.39, <i>p</i> = 0.01) after placebo, an association eliminated by arginine supplementation (<i>r</i> = −0.04, <i>p</i> = 0.70). A dose-dependent increase in platelet-mitochondrial activity occurred after arginine versus no change after placebo (<i>p</i> < 0.001); plasma protein-carbonyl levels, a measure of oxidative stress, decreased after arginine therapy (<i>p</i> < 0.001) but increased in the placebo group (<i>p</i> = 0.02). SCD-VOE is associated with an acquired arginine deficiency that correlates with worse clinical outcomes. Arginine improved mitochondrial function and decreased oxidative stress compared to placebo, with clinically relevant opioid-sparing becoming significant in children with the lowest arginine concentration.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Trial Registration</h3>\u0000 \u0000 <p>Registered with ClinicalTrials.gov (NCT02536170) in August 2015</p>\u0000 </section>\u0000 </div>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 7","pages":"1119-1131"},"PeriodicalIF":10.1,"publicationDate":"2025-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143866820","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Daniel Wang, Manraj Sra, Samira Glaeser-Khan, Daniel Y. Wang, Ranya Moshashaian-Asl, Satoko Ito, Adam Cuker, George Goshua
{"title":"Cost-Effectiveness of Ferritin Screening Thresholds for Iron Deficiency in Reproductive-Age Women","authors":"Daniel Wang, Manraj Sra, Samira Glaeser-Khan, Daniel Y. Wang, Ranya Moshashaian-Asl, Satoko Ito, Adam Cuker, George Goshua","doi":"10.1002/ajh.27686","DOIUrl":"10.1002/ajh.27686","url":null,"abstract":"<p>Iron deficiency (ID) is a top five leading cause of disability-adjusted life-years in women of reproductive age around the world. Despite its enormous health burden, no screening guidelines exist for the detection and treatment of ID in women of reproductive age. We sought to determine the cost-effectiveness of screening versus no screening for ID in women of reproductive age in the United States. A lifetime simulation of women of reproductive age was conducted using a Markov cohort model under three strategies: (1) no screening, (2) screening at a ferritin threshold of 15 μg/L, and (3) screening at a ferritin threshold of 25 μg/L, from the US health system perspective, and at a willingness-to-pay threshold of $100 000/quality-adjusted life year (QALY). Epidemiologically informed ID prevalence estimates sourced from the National Health and Nutrition Examination Survey were employed for model parameterization. The primary outcome was the incremental cost-effectiveness ratio (ICER, in $/QALY). Base-case results for the three strategies accrued $209 700, $210 200, and $210 200 discounted lifetime costs and 23.6, 24.0, and 24.4 discounted lifetime QALYs, respectively. Screening at a ferritin threshold of 25 μg/L was the cost-effective intervention with an ICER of $680/QALY (95% credible interval $350–$750/QALY). In dual base-case analyses examining intravenous rather than oral iron repletion for treatment, screening at a ferritin threshold of 25 μg/L remained the cost-effective intervention with an ICER of $2300/QALY (95% CI $1800–$3800/QALY). In probabilistic sensitivity analyses, screening at a ferritin threshold of 25 μg/L was the cost-effective intervention in 100% of 10 000 second order Monte Carlo iterations.</p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 7","pages":"1132-1140"},"PeriodicalIF":10.1,"publicationDate":"2025-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.27686","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143837018","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Fateeha Furqan, Kwang W. Ahn, Manmeet Kaur, Jinalben Patel, Stephen Ansell, Farrukh T. Awan, John Baird, Evandro Bezerra, Umar Farooq, Henry Fung, Arushi Khurana, Lazaros Lekakis, Forat Lutfi, John McCarty, Akash Mukherjee, Rajneesh Nath, Jason Romancik, Stephen J. Schuster, Melody Smith, Allison Winter, Cameron Turtle, Craig Sauter, Mazyar Shadman, Alex Herrara, Mehdi Hamadani
{"title":"Autologous Transplant or CAR-T as Consolidation Options in MYC Rearranged Large B-Cell Lymphoma Patients in Remission After Salvage Treatments","authors":"Fateeha Furqan, Kwang W. Ahn, Manmeet Kaur, Jinalben Patel, Stephen Ansell, Farrukh T. Awan, John Baird, Evandro Bezerra, Umar Farooq, Henry Fung, Arushi Khurana, Lazaros Lekakis, Forat Lutfi, John McCarty, Akash Mukherjee, Rajneesh Nath, Jason Romancik, Stephen J. Schuster, Melody Smith, Allison Winter, Cameron Turtle, Craig Sauter, Mazyar Shadman, Alex Herrara, Mehdi Hamadani","doi":"10.1002/ajh.27687","DOIUrl":"10.1002/ajh.27687","url":null,"abstract":"<div>\u0000 \u0000 <p>Although recent studies have demonstrated the efficacy of chimeric antigen receptor T-cell (CAR-T) therapy in relapsed large B-cell lymphoma (LBCL) with MYC rearrangement (R-MYC), the data comparing CAR-T to autologous hematopoietic cell transplant (auto-HCT) in such patients who achieve a complete or partial response (CR/PR) after salvage therapies are limited. We compared the clinical outcomes of patients with R-MYC LBCL (including double and triple hit lymphomas) who underwent CAR-T or auto-HCT after achieving a CR/PR with salvage therapies using the Center for International Blood & Marrow Transplant Research registry. Among the 252 patients (auto-HCT = 98, CAR-T = 154), relative to auto-HCT, CAR-T was associated with significantly lower overall survival (OS) (Hazard Ratio [HR] 2.09, 95% CI 1.38–3.15, <i>p</i> < 0.001) on multivariate analysis. There were no differences in progression-free survival (PFS) (HR 1.21, 95% CI 0.81–1.8 <i>p</i> = 0.36), risk of relapse (HR 1.1, 95% CI 0.71–1.69 <i>p</i> = 0.68), nonrelapse mortality (NRM) (HR 1.74, 95% CI 0.64–4.7 <i>p</i> = 0.28) while the post-relapse survival was longer in auto-HCT relative to CAR-T (HR 1.93, 95% CI 1.21–3.06 <i>p</i> = 0.01). On propensity score matched analysis accounting for differences in characteristics across the two cohorts, we detected no significant differences in OS (HR 1.72, 95% CI 0.92–3.21 <i>p</i> = 0.09), PFS (HR 1.04, 95% CI 0.64–1.68 <i>p</i> = 0.88), NRM (HR 1.22, 95% CI 0.35–4.2 <i>p</i> = 0.76), relapse (HR = 0.93, 95% CI 0.54–1.6 <i>p</i> = 0.8) and post-relapse survival (HR 2.25, 95% CI 0.98–5.17, <i>p</i> = 0.06). These data, although retrospective, support consideration for auto-HCT in patients with R-MYC LBCL who achieve a CR/PR after salvage therapies, particularly in regions with no or limited access to CAR-T.</p>\u0000 </div>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 7","pages":"1152-1162"},"PeriodicalIF":10.1,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143832014","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Eli Muchtar, Vaishali Sanchorawala, Hamza Hassan, Ute Hegenbart, Stefan Schönland, Hans C. Lee, Muzaffar Qazilbash, Andrew Kin, Jeffrey Zonder, Eapen Jacob, Francis Buadi, Angela Dispenzieri, David Dingli, Sally Arai, Michelle Chin, Rajshekhar Chakraborty, Suzanne Lentzsch, Hila Magen, Eden Shkury, Caitlin Sarubbi, Heather Landau, Morie Gertz
{"title":"The Impact of Melphalan Conditioning and CD34\u0000 + Cell Dose and Schedule on Post-Transplant Outcomes in AL Amyloidosis","authors":"Eli Muchtar, Vaishali Sanchorawala, Hamza Hassan, Ute Hegenbart, Stefan Schönland, Hans C. Lee, Muzaffar Qazilbash, Andrew Kin, Jeffrey Zonder, Eapen Jacob, Francis Buadi, Angela Dispenzieri, David Dingli, Sally Arai, Michelle Chin, Rajshekhar Chakraborty, Suzanne Lentzsch, Hila Magen, Eden Shkury, Caitlin Sarubbi, Heather Landau, Morie Gertz","doi":"10.1002/ajh.27685","DOIUrl":"10.1002/ajh.27685","url":null,"abstract":"<div>\u0000 \u0000 <p>The optimal conditioning schedule and CD34+ cell dose for autologous stem cell transplantation (ASCT) for AL amyloidosis is unknown. Patients (<i>n</i> = 1704) who underwent ASCT for AL amyloidosis between 2003 and 2020 in 9 centers were included. Data on melphalan conditioning dose, number of conditioning days, whether a rest day between conditioning and stem cell infusion was given or not, and infused CD34<sup>+</sup> cell dose were collected. Full-dose melphalan (≥ 180 mg/m<sup>2</sup>) was administered in 63.7% of the patients, and 80.4% had melphalan split into 2-day conditioning. A rest day (day −1) between the conditioning regimen and stem cell infusion was provided in 52.5% of patients. The median infused CD34<sup>+</sup> cell count was 4.7 × 10<sup>6</sup>/kg. An infused CD34<sup>+</sup> cell count ≥ 4.5 × 10<sup>6</sup>/kg was associated with a shorter time to neutrophil and platelet engraftment. In a nominal regression analysis, full-dose melphalan, 1-day melphalan conditioning, and omitting a rest day between conditioning and stem cell infusion were independent predictors of post-ASCT higher deep hematological response. The median follow-up was 8.6 years, and 38% of patients died. Independent predictors of superior overall survival in multivariate Cox regression analysis included full-dose melphalan, having no rest day, and infused CD34<sup>+</sup> cells ≥ 4.5 × 10<sup>6</sup>/kg. Independent predictors of higher day-100 transplant-related mortality (TRM) in nominal logistic regression analysis included poorer performance status, NT-proBNP/BNP ≥ 1800/400 pg/mL, serum albumin < 2.5 g/dL, CD34<sup>+</sup> cells < 4.5 × 10<sup>6</sup>/kg, and not having a rest day. In conclusion, 1 day of melphalan conditioning and administration of CD34<sup>+</sup> cells ≥ 4.5 × 10<sup>6</sup>/kg are recommended for ASCT in AL amyloidosis.</p>\u0000 </div>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 7","pages":"1141-1151"},"PeriodicalIF":10.1,"publicationDate":"2025-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143819983","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}