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doi:10.1002/ajh.v94.2

Cited by 3 publications

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“…The combination of BZ, dexamethasone, and RTX was given to 23 untreated patients, with administration of intravenous BZ at 1.3 mg/m 2 , dexamethasone at 40 mg twice per week on days 1, 4, 8, and 11, and RTX 375 mg/m 2 on day 11 for four cycles as induction treatment and four more cycles at 3 months as maintenance treatment. 4,15 The overall response rate (ORR) and major response rate (RR) were 96% and 83%, respectively, with a median time in target range (TTR) of 1.4 months. Sixty percent of patients discontinued treatment after four cycles because of treatment-related peripheral neuropathy.…”

Section: Discussionmentioning

confidence: 99%

“…2,3 Nevertheless, some hemorrhagic signs and nasal bleeding also have been related to hyperviscocity syndrome. 4 From the clinical point of view, AVWS associated with WM is a challenging condition, particularly when severe bleeding is observed at diagnosis. Emergency therapy is necessary as well as the rapid initiation of a treatment for the underlying condition although it is not always associated with a good and rapid response to the bleeding.…”

Section: Introductionmentioning

confidence: 99%

“…Moreover, when a suspected hyperviscocity syndrome is suspected, the use of RTX can be associated with a flare syndrome. 4 Based on our favorable experience with one patient with AVWS associated with an IgG-monoclonal gammopathy of undetermined significance (MGUS) that was refractory to RTX and who responded successfully to a bortezomib (BZ)based therapy, 5 in this study we similarly treated two other patients with WM who presented a concomitant severe AVWS (Table 1). These cases were refractory to several cycles of the chemo-immunotherapy regimen R-CVP21 (RTX 375 mg/m 2 /day × 1 day; Cyclophosphamide 750 mg/m 2 /day × 1 day; Vincristine 1.4 mg/m 2 /day × 1 day, and methylprednisolone 60 mg/m 2 /day × 5 days).…”

Section: Introductionmentioning

confidence: 99%

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Good Profile of Efficacy/Tolerance of Bortezomib or Idelalisib in Waldenström Macroglobulinemia Associated with Acquired Von Willebrand Syndrome

Ojeda-Uribe¹,

Rimelen²,

Marzullo³

2020

JBM

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Acquired von Willebrand syndrome (AVWS) in the setting of Waldenström macroglobulinemia (WM) is a challenging condition. No real standard of care is recommended for these patients, although the therapeutic strategy should include a rapid approach to the emergency bleeding events and to the underlying malignant lymphoid disorder. We report here our experience treating three elderly patients with these concomitant hematologic entities. The use of a bortezomib-based chemotherapy regimen showed a good profile of tolerance and efficacy even in a long-term follow-up period. These patients were treated for several years before switching their therapy to idelalisib, a targeted oral therapy that inhibits phosphatidylinositol 3-kinase isoform-delta (PI3KD), which is part of the signaling pathway downstream B-cell receptor. This approach was well tolerated and efficacious, although some adverse effects were observed, particularly at hepatic levels, but were all reversible. The same profile of tolerance/efficacy was observed in one very old patient who received idelalisib as a first-line therapy. We think that bortezomib-based therapy could be considered in refractory patients with AVWS associated with WM.

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Good Profile of Efficacy/Tolerance of Bortezomib or Idelalisib in Waldenström Macroglobulinemia Associated with Acquired Von Willebrand Syndrome

Ojeda-Uribe¹,

Rimelen²,

Marzullo³

2020

JBM

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“…Up to 50% of adults with ALL have persistently detectable MRD after initial chemotherapy, which is highly predictive of eventual overt hematologic relapse. [42][43][44] After the early clinical development of blinatumomab was hampered by significant treatment-emergent adverse effects thought to be related to high baseline tumor burden, it was hypothesized that blinatumomab may be efficacious with fewer instances of high-grade CRS in the setting of MRD-only disease. In a phase II study, 21 patients with MRD-positive ALL in CR received blinatumomab dosed at 15 mcg/m 2 /day for 4 weeks followed by a 2-week drugfree period.…”

Section: Mrd-positive B-cell Allmentioning

confidence: 99%

Blinatumomab for the Treatment of Adult B-Cell Acute Lymphoblastic Leukemia: Toward a New Era of Targeted Immunotherapy

Franquiz¹,

Short²

2020

BTT

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Several therapeutic advancements in the treatment of B-cell acute lymphoblastic leukemia (ALL) have surfaced in the past decade, primarily driven by an increased understanding of the immunopathobiology of this disease. The clinical use of blinatumomab, a bispecific antibody that coordinates cytotoxic CD3+ T lymphocytes and CD19+ lymphoblasts, has resulted in improved outcomes in both relapsed/refractory and minimal residual disease-positive B-cell ALL. Promising emerging data also demonstrate the efficacy of this agent in the frontline setting and in combination regimens. Uncertainty remains regarding the optimal sequencing and combination of blinatumomab with cytotoxic chemotherapy and other emerging agents. The pharmacology and clinical data on blinatumomab for adult B-cell ALL, both as monotherapy and in combinations, will be reviewed herein.

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Severe Maternal Morbidity and Mortality in Sickle Cell Disease in the National Inpatient Sample, 2012-2018

et al. 2023

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ImportancePregnancy outcomes are historically poor among people with sickle cell disease (SCD) in the US, most of whom have Black race. Whether outcomes have improved is unknown.ObjectiveTo tabulate adverse pregnancy outcomes among patients with SCD, comparing outcomes of deliveries among Black people with SCD with those of Black people without SCD and a control non-Black population, and to measure the association of racial disparities with adverse outcomes in SCD pregnancies.Design, Setting, and ParticipantsThis cross-sectional study was a secondary analysis involving data from National Inpatient Sample, a nationally representative sample of 20% of acute hospital admissions in the US, between 2012 and 2018. The data set included all admissions with codes for delivery of a pregnancy among people aged 11 to 55 years. Data were analyzed from September 2021 to August 2022.ExposuresSCD, racial disparities.Main Outcomes and MeasuresSevere maternal morbidity (SMM) as measured by the US Centers for Disease Control and Prevention’s index alongside other outcomes; multiple logistic regression was used to compare the odds for adverse pregnancy outcomes.ResultsThe sample included 5 401 899 deliveries, including 3901 deliveries among people with SCD and 742 164 deliveries among people with Black race. Compared with the non-Black control group, patients with SCD and Black patients were younger (mean [SD] age: SCD, 27.2 [5.9] years; Black, 27.1 [6.1] years vs 28.7 [5.9] years) and more likely to have public insurance (SCD, 2609 deliveries [67.3%]; Black, 496 828 deliveries [65.4%] vs 1 880 198 deliveries [40.8%]). The maternal mortality rate in deliveries among people with SCD was 26 times greater than in the non-Black control group and more than 10 times greater than among Black pregnant people without SCD (Per 10 000 deliveries: SCD 13.3; 95% CI, 5.7-31.2; Black race, 1.2; 95% CI, 1.0-1.5; non-Black control 0.5; 95% CI, 0.5-0.6). Compared with the control group, SCD deliveries had higher odds of SMM (adjusted odds ratio [aOR], 7.22; 95% CI, 6.25-8.34; P &lt; .001), especially cerebrovascular events (aOR, 22.00; 95% CI, 15.25-31.72; P &lt; .001) and thromboembolism (aOR, 17.34; 95% CI, 11.55-26.03; P &lt; .001). Racial disparities explained a median (IQR) 28.9% (21.2%-33.1%) of the increased risk in deliveries to people with SCD and between 40% and 50% of the increased risk for acute kidney failure (excess risk [ER], 56.9%; 95% CI, 54.3%-59.3%), intrauterine fetal demise (ER, 47.8%; 95% CI, 46.6%-49.1%), and eclampsia (ER, 42.1%; 95% CI, 37.9%-46.1%).Conclusions and RelevanceIn this large cross-sectional study of pregnancy outcomes in people with SCD, the risk for SMM was higher compared with deliveries among people without SCD, especially for thrombotic events, organ failure, and death. Racial disparities were associated with adverse outcomes. Our findings compel scientific, clinical, and political effort to improve outcomes for pregnant people with SCD.

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Cited by 3 publications

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<p>Good Profile of Efficacy/Tolerance of Bortezomib or Idelalisib in Waldenström Macroglobulinemia Associated with Acquired Von Willebrand Syndrome</p>

<p>Good Profile of Efficacy/Tolerance of Bortezomib or Idelalisib in Waldenström Macroglobulinemia Associated with Acquired Von Willebrand Syndrome</p>

<p>Blinatumomab for the Treatment of Adult B-Cell Acute Lymphoblastic Leukemia: Toward a New Era of Targeted Immunotherapy</p>

Severe Maternal Morbidity and Mortality in Sickle Cell Disease in the National Inpatient Sample, 2012-2018

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