Publications by authors named "Zachariah DeFilipp"

Article Synopsis
  • * Researchers analyzed data from 450 patients, finding that 310 were classified as ultra-low risk (ULR) based on their rapid clinical response and low MAP scores, leading to significantly better outcomes.
  • * Patients in the ULR group had higher response rates at day 28 and lower non-relapse mortality at six months, suggesting that careful monitoring can guide safer, more effective GVHD treatment strategies.
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  • * A study analyzing 667 bone marrow harvests between 1999 and 2021 found a 13% contamination rate, primarily from common skin bacteria, with male donors showing significantly higher contamination rates (18% vs. 6.8% for females).
  • * The research is notable for being the largest single-center study to explore contamination rates and highlights male donor sex as a key factor, potentially due to differences in skin flora.
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The objective of this meta-analysis (MA) was to evaluate the efficacy and safety of extracorporeal photopheresis (ECP) for the treatment of steroid-refractory chronic graft-versus-host disease (SR-cGvHD). A systematic literature review (SLR) was conducted according to PRISMA guidelines, followed by a feasibility assessment (FA) to assess potential between-study heterogeneity in the meta-analysis (MA). Random-effects MAs were performed for overall survival (OS), failure-free survival (FFS), overall response rate (ORR) and skin-specific response.

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To examine activity of ibrutinib in steroid-refractory chronic GVHD (SR-cGVHD) after FDA approval, we conducted a multicenter retrospective study. Data were standardly collected (N=270 from 19 centers). Involved organs included skin (75%), eye (61%), mouth (54%), joint/fascia (47%), GI (26%), lung (27%), liver (19%), genital (7%), other (4.

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  • - Bronchiolitis obliterans syndrome (BOS) is a serious lung condition that can develop after receiving a stem cell transplant, and this study tested the effectiveness of ruxolitinib in treating it.
  • - The trial involved 49 adult participants divided into two groups based on how recently they were diagnosed with BOS; results showed that 27.8% of newly diagnosed patients improved, while 92.3% of those with established BOS maintained stable lung function after treatment.
  • - Overall, ruxolitinib had a lung-specific response rate of 34.7% among all participants with most positive outcomes seen in patients with mild to moderate disease, indicating it could be a beneficial treatment option for BOS
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  • * A panel of experts made recommendations emphasizing the use of bone marrow as a graft source and preferred rabbit antithymocyte globulin over horse ATG for conditioning. They also support using fludarabine for high-risk patients and expanding HCT eligibility to fit older adults.
  • * The panel advocates prioritizing matched unrelated or haploidentical donor transplants over immunosuppressive therapy when a matched related donor isn't available and suggests specific GVHD prophylaxis options for donor
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  • The study investigates the effectiveness of axatilimab, a CSF1R-blocking antibody, for treating chronic graft-versus-host disease (GVHD) in patients post-hematopoietic stem-cell transplantation.
  • In a phase 2 trial involving 241 participants, different doses of axatilimab were tested, with overall response rates of 74%, 67%, and 50% across three dose groups.
  • Alongside substantial improvement in GVHD symptoms measured by patient-reported outcomes, the most frequent side effects were temporary lab abnormalities linked to the treatment.
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Hematopoietic stem cell transplant (HSCT) survivors frequently experience persistent sexual dysfunction, which is associated with impaired quality of life and increased psychological distress. The lack of availability of clinicians with expertise in sexual health limits the capacity to address sexual health concerns in HSCT survivors. Digital health applications may offer a patient-centered and scalable solution to address sexual health concerns in cancer survivors.

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  • - The study analyzed how different types of donors affect outcomes of hematopoietic cell transplantation (HCT) in patients with myelofibrosis, finding that the use of haploidentical donors rose significantly from 3% in 2013 to 19% in 2019.
  • - Among 1,032 patients with chronic-phase myelofibrosis, matched sibling donor HCTs showed better overall survival in the first three months compared to haploidentical and matched unrelated donor HCTs, with notably lower rates of graft failure.
  • - While matched sibling donors had superior early outcomes, there were no significant differences in long-term survival or disease-free survival among the different donor types, suggesting hap
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Allogeneic hematopoietic cell transplantation (HCT) survivors may benefit from routine screening for post-transplant complications. However, the impact of formal survivorship efforts to promote screening adherence is uncertain. The effect of a formal HCT survivorship program to promote screening adherence was evaluated.

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The significance of biomarkers in second-line treatment for acute graft-versus-host disease (GVHD) has not been well characterized. We analyzed clinical data and serum samples at the initiation of second-line systemic treatment of acute GVHD from 167 patients from 17 centers of the Mount Sinai Acute GVHD International Consortium (MAGIC) between 2016 and 2021. Sixty-two patients received ruxolitinib-based therapy, whereas 102 received other systemic agents.

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Graft-versus-host disease (GVHD) is a major cause of nonrelapse mortality (NRM) after allogeneic hematopoietic cell transplantation. Algorithms containing either the gastrointestinal (GI) GVHD biomarker amphiregulin (AREG) or a combination of 2 GI GVHD biomarkers (suppressor of tumorigenicity-2 [ST2] + regenerating family member 3 alpha [REG3α]) when measured at GVHD diagnosis are validated predictors of NRM risk but have never been assessed in the same patients using identical statistical methods. We measured the serum concentrations of ST2, REG3α, and AREG by enzyme-linked immunosorbent assay at the time of GVHD diagnosis in 715 patients divided by the date of transplantation into training (2004-2015) and validation (2015-2017) cohorts.

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Article Synopsis
  • Chimeric antigen receptor T cell therapy (CAR-T) has significantly improved treatment for relapsed and/or refractory multiple myeloma (RRMM), but treatment failures remain common, posing major challenges in patient management.
  • An online survey, conducted by the American Society for Transplantation and Cellular Therapy (ASTCT) Committee on Practice Guidelines, gathered insights from 80 physicians regarding their practices for monitoring and addressing CAR-T failures in RRMM patients.
  • The survey revealed variations across treatment centers, particularly in post-CAR-T evaluation practices and available rescue therapies, highlighting the need for collaborative research and established clinical guidelines to improve patient outcomes.
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  • Acute graft versus host disease (GVHD) is a significant complication for children undergoing hematopoietic cell transplantation (HCT), and new tools have been developed to assess the risk of nonrelapse mortality (NRM) at the onset of treatment.
  • The study compared the Minnesota risk system and the MAGIC algorithm using biomarkers ST2 and REG3α, finding that MAGIC's probabilities (MAPs) were more effective in predicting outcomes in pediatric patients compared to Minnesota risk alone.
  • A combined model using both Minnesota risk and biomarker scores increased prediction accuracy further, demonstrating the potential for more personalized treatment approaches for children with GVHD.
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Disruption of the intestinal microbiome is observed with acute graft-versus-host disease (GVHD) of the lower gastrointestinal (LGI) tract, and fecal microbiota transplantation (FMT) has successfully cured steroid-refractory cases. In this open-label, single-arm, pilot study, third-party, single-donor FMT was administered in combination with systemic corticosteroids to participants with high-risk acute LGI GVHD, with a focus on treatment-naïve cases. Participants were scheduled to receive 1 induction dose (15 capsules per day for 2 consecutive days), followed by 3 weekly maintenance doses, consisting of 15 capsules per dose.

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Background: More than half the long-term survivors of allogeneic hematopoietic cell transplantation develop chronic graft-versus-host disease (GVHD), a debilitating inflammatory syndrome. Supportive interventions to assist survivors in coping with chronic GVHD are critically needed.

Patients And Methods: We conducted a pilot randomized clinical trial of a multidisciplinary group intervention (Horizons Program; n=39) versus minimally enhanced usual care (n=41) for patients with moderate or severe chronic GVHD.

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The absence of a standardized definition for graft-versus-host disease (GVHD) flares and data on its clinical course are significant concerns. We retrospectively evaluated 968 patients across 23 Mount Sinai Acute GVHD International Consortium (MAGIC) transplant centers who achieved complete response (CR) or very good partial response (VGPR) within 4 weeks of treatment. The cumulative incidence of flares within 6 months was 22%, and flares were associated with a higher risk of nonrelapse mortality (NRM; adjusted hazard ratio [aHR], 4.

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The overall response rate (ORR) 28 days after treatment has been adopted as the primary endpoint for clinical trials of acute graft versus host disease (GVHD). However, physicians often need to modify immunosuppression earlier than day (D) 28, and non-relapse mortality (NRM) does not always correlate with ORR at D28. We studied 1144 patients that received systemic treatment for GVHD in the Mount Sinai Acute GVHD International Consortium (MAGIC) and divided them into a training set (n=764) and a validation set (n=380).

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Background: Patients with hematologic malignancies undergoing hematopoietic stem cell transplantation (HSCT) commonly experience debilitating physical and psychological symptoms during a 3-4-week-hospitalization. During hospitalization, caregivers (i.e.

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  • Acute kidney injury (AKI) is common in critically ill patients after allogeneic hematopoietic stem cell transplantation (allo-HSCT), with a study revealing that 21% of 529 patients required kidney replacement therapy (AKI-KRT).
  • Risk factors for needing AKI-KRT include lower kidney function before transplant, certain complications like veno-occlusive disease, and critical health indicators upon ICU admission.
  • A striking 87.4% of patients undergoing AKI-KRT died within 90 days, emphasizing the need for careful evaluation before initiating KRT in these patients.
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The study aimed to determine the association of chronic graft-versus-host disease (cGVHD) diagnosis and severity with the development of subsequent neoplasms (SN) and nonmalignant late effects (NM-LE) in 2-year disease-free adult survivors following hematopoietic cell transplantation (HCT) for a hematologic malignancy. To do so, we conducted a retrospective analysis of 3884 survivors of HCT for hematologic malignancy in the Center of International Blood and Marrow Transplant Research database. We conducted a landmark analysis at the 2-year post-transplantation date, comparing first SN and NM-LE in survivors with and without cGVHD.

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Severe acute graft-versus-host disease (aGVHD) is associated with significant mortality and morbidity, especially in steroid-resistant (SR) cases. Spatial transcriptomic technology can elucidate tissue-based interactions in vivo and possibly identify predictors of treatment response. Tissue sections from 32 treatment-naïve patients with biopsy-confirmed lower gastrointestinal (GI) aGVHD were obtained.

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