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ACCELERATE: A Patient-Powered Natural History Study Design Enabling Clinical and Therapeutic Discoveries in a Rare Disorder.

Geographically dispersed patients, inconsistent treatment tracking, and limited infrastructure slow research for many orphan diseases. We assess the feasibility of a patient-powered study design to overcome these challenges for Castleman disease, a rare hematologic disorder. Here, we report initial results from the ACCELERATE natural history registry. ACCELERATE includes a traditional physician-reported arm and a patient-powered arm, which enables patients to directly contribute medical data and biospecimens. This study design enables successful enrollment, with the 5-year minimum enrollment g

Case Report: Endothelial-targeted bridging therapy for a TTP-like phenotype in fulminant iMCD-TAFRO.

BACKGROUND: iMCD-TAFRO (the TAFRO clinical subtype of idiopathic multicentric Castleman disease (iMCD)) is characterized by thrombocytopenia, anasarca, fever, reticulin fibrosis, and organomegaly. Diagnosis is challenging due to its rarity, non-specific early presentation, and overlap with sepsis, lymphoma, and thrombotic microangiopathy (TMA). Endothelial injury is increasingly recognized as a central driver of its severe complications. CASE PRESENTATION: A previously healthy 20-year-old woman presented with rapid progression of fever, anasarca, jaundice, and respiratory failure. Laboratory

Prevalence of functional iron deficiency in patients with malignancies and its impact on fatigue-specific quality of life.

BackgroundFunctional iron deficiency (FID) as defined by the European Society for Medical Oncology (ESMO) occurs in patients with malignant neoplasms and might be a target to improve fatigue-specific quality of life (QoL). The prevalence of FID in that population, especially in patients with a haemoglobin (Hb) of 10-11 g/dl and Hb > 11 g/dl, is unclear. The aim of this study was to obtain data on the prevalence of FID in patients with solid tumours and haematological malignancies independent of Hb and age, and to investigate the potential of FID as a target to improve fatigue-specific QoL.Pati

CXCR4-directed endoradiotherapy with [177Lu]Pentixather added to total body irradiation for myeloablative conditioning in patients with relapsed/refractory acute myeloid leukemia.

Rationale: Despite recent advances in the targeted therapy of AML, the disease continues to have a poor prognosis. Allogeneic hematopoietic stem cell transplantation (alloSCT) remains to be the curative therapy option for fit patients with high-risk disease. Especially patients with relapsed or refractory (r/r) AML continue to have poor outcomes. Myeloablative total body irradiation (TBI) based conditioning can be used in AML patients refractory to multiple lines of standard therapy, but the optimal conditioning regimen remains unclear for patients considered to be chemotherapy- refractory. Fe

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… Integrating CXCR4-directed ERT into auto- or alloSCT strategies for heavily pretreated lymphoproliferative disorders, including lymphomas and multiple myeloma, has achieved remissions in all evaluated patients 33 , 38 - 40 . …

Hematopoietic progenitor cell mobilization for autologous transplantation - a literature review.

The use of high-dose chemotherapy with autologous support of hematopoietic progenitor cells is an effective strategy to treat various hematologic neoplasms, such as non-Hodgkin lymphomas and multiple myeloma. Mobilized peripheral blood progenitor cells are the main source of support for autologous transplants, and collection of an adequate number of hematopoietic progenitor cells is a critical step in the autologous transplant procedure. Traditional strategies, based on the use of growth factors with or without chemotherapy, have limitations even when remobilizations are performed. Granulocyte

Update on the role of autologous hematopoietic stem cell transplantation in multiple myeloma.

Autologous stem cell transplantation is considered the standard of care for multiple myeloma patients aged < 65 years with no relevant comorbidities. The addition of drugs acting both on bone marrow microenvironment and on neoplastic plasma cells has significantly increased the proportion of patients achieving a complete remission after induction therapy, and these results are mantained after high-dose melphalan, leading to a prolonged disease control. Studies are being carried out in order to evaluate whether short term consolidation or long-term maintenance therapy can result into disease er

Glucosylsphingosine (Lyso-Gb1) as a reliable biomarker in Gaucher disease: a narrative review.

BACKGROUND: Gaucher disease (GD) is a rare, inherited, autosomal recessive disorder caused by a deficiency of the lysosomal enzyme, acid β-glucosidase. Its diagnosis is achieved via measurements of acid β-glucosidase activity in either fresh peripheral blood leukocytes or dried blood spots, and confirmed by identifying characteristic mutations in the GBA1 gene. Currently, several biomarkers are available for disease monitoring. Chitotriosidase has been used over the last 20 years to assess the severity of GD, but lacks specificity in GD patients. Conversely, the deacylated form of glucosylcera

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… Furthermore, lyso-Gb1 has an important role in the pathogenetic mechanism of PD due to its cerebral accumulation, and in B-cell lymphoproliferative disorders, such as multiple myeloma, due to humoral immunity dysregulation by chronic antigenic stimulation. …

A comparative analysis of transcriptomics of newly diagnosed multiple myeloma: exploring drug repurposing.

Multiple myeloma (MM) is an incurable malignant plasma cell disorder characterized by the infiltration of clonal plasma cells in the bone marrow compartment. Gene Expression Profiling (GEP) has emerged as a powerful investigation tool in modern myeloma research enabling the dissection of the molecular background of MM and allowing the identification of gene products that could potentially serve as targets for therapeutic intervention. In this study we investigated shared transcriptomic abnormalities across newly diagnosed multiple myeloma (NDMM) patient cohorts. In total, publicly available tr

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… The progression risk from MGUS to symptomatic MM or other lymphoproliferative disorders accounts for 1% per year ( 4 ). Hallmark features of symptomatic MM include anemia, hypercalcemia, renal insufficiency, osteolytic bone lesions and increased vulnerability to infections ( 5 ). …

Hematopoietic stem cell transplantation.

More than 25,000 hematopoietic stem cell transplantations (HSCTs) are performed each year for the treatment of lymphoma, leukemia, immune-deficiency illnesses, congenital metabolic defects, hemoglobinopathies, and myelodysplastic and myeloproliferative syndromes. Before transplantation, patients receive intensive myeloablative chemoradiotherapy followed by stem cell "rescue." Autologous HSCT is performed using the patient's own hematopoietic stem cells, which are harvested before transplantation and reinfused after myeloablation. Allogeneic HSCT uses human leukocyte antigen (HLA)-matched stem

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… CMV infection and other transplant-related diseases, such as EBV-related lymphoproliferative disorders, can be treated and prevented by adoptive cellular transfer therapy. …

Poster Abstracts

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Post-Transplant Cyclophosphamide and Tacrolimus-Mycophenolate Mofetil Combination Governs GVHD and Immunosuppression Need, Reducing Late Toxicities in Allogeneic Peripheral Blood Hematopoietic Cell Transplantation from HLA-Matched Donors.

Combined direct antineoplastic activity and the long-lasting immunological effects of allogeneic hematopoietic cell transplant (HCT) can cure many hematological malignancies, but broad adoption requires non-relapse mortality (NRM) rates and graft-versus-host disease (GVHD) control. Recently, posttransplant cyclophosphamide (PTCy) given after a bone marrow transplant significantly reduced GVHD-incidence, while PTCy given with tacrolimus/mofetil mycophenolate (T/MMF) showed activity following allogeneic peripheral blood stem cell transplantation (alloPBSCT). Here, we report the experience of a l

High Mortality of HLH in ICU Regardless Etiology or Treatment.

Background: Adult hemophagocytic lymphohistiocytosis (HLH) is highly lethal in the ICU. The diagnostic and therapeutic emergency that HLH represents is compounded by its unknown pathophysiological mechanisms. Here, we report on a large cohort of adult HLH in the ICU (ICU-HLH). We analyzed prognostic factors associated with mortality to define the diagnostic and therapeutic challenges in this specific population. Methods: This retrospective study included adult patients diagnosed with HLH in four ICUs in Marseille, France between 2010 and 2020. Patients who fulfilled the HLH-2004 criteria (≥ 4/

A multicenter prospective study of 515 febrile neutropenia episodes in Argentina during a 5-year period.

For better management of patients with febrile neutropenia, our study investigated the epidemiologic, microbiologic, and clinical characteristics of adult inpatients with febrile neutropenia and their mortality-associated factors. To this end, we carried out a prospective, observational, multicenter study in 28 Argentinian hospitals between 2007 and 2012. We included 515 episodes of febrile neutropenia from 346 patients, median age 49 years. Neutropenia followed chemotherapy in 77% of cases, half of the cases due to hematological malignancies. Most episodes were classified as high-risk accordi

The Risk of Sarcoidosis Misdiagnosis and the Harmful Effect of Corticosteroids When the Disease Picture Is Incomplete.

Sarcoidosis is a complex granulomatous disease of unknown etiology. Due to the heterogeneity of the disease, the diagnosis remains challenging in many cases, often at the physician's discretion, requiring a thorough and complex investigation. Many other granulomatous diseases have the potential to mimic sarcoidosis, whether infectious, occupational, or autoimmune diseases and starting an unnecessary corticosteroid treatment can worsen the patient's prognosis, leading to side effects that can be harder to treat than the actual disease.

Review of the Longitudinal Management of Autoimmune Encephalitis, Potential Biomarkers, and Novel Therapeutics.

Purpose of reviewIncreasing awareness and earlier diagnosis of autoimmune encephalitis (AE) have led to a greater number of patients being cared for longitudinally by neurologists. Although many neurologists are now familiar with the general approach to diagnosis and acute immunosuppression, this review aims to provide neurologists with guidance related to management beyond the acute phase of disease, including long-term immunosuppression, monitoring, potential biomarkers of disease activity, outcome measures, and symptom management.Recent findingsObservational studies in AE have demonstrated

Iceland screens, treats, or prevents multiple myeloma (iStopMM): a population-based screening study for monoclonal gammopathy of undetermined significance and randomized controlled trial of follow-up strategies.

Monoclonal gammopathy of undetermined significance (MGUS) precedes multiple myeloma (MM). Population-based screening for MGUS could identify candidates for early treatment in MM. Here we describe the Iceland Screens, Treats, or Prevents Multiple Myeloma study (iStopMM), the first population-based screening study for MGUS including a randomized trial of follow-up strategies. Icelandic residents born before 1976 were offered participation. Blood samples are collected alongside blood sampling in the Icelandic healthcare system. Participants with MGUS are randomized to three study arms. Arm 1 is n

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… IgM MGUS is caused by monoclonal lymphoplasmacytic lymphocytes and is a precursor to other lymphoproliferative disorders (LP), most notably Waldenström’s macroglobulinemia (WM), and rarely MM 3 . …

Acute Kidney Injury in Monoclonal Gammopathies.

Monoclonal gammopathies (MG) encompass a variety of disorders related to clonal expansion and/or malignant transformation of B lymphocytes. Deposition of free immunoglobulin (Ig) components (light or heavy chains, LC/HC) within the kidney during MG may result over time in multiple types and degrees of injury, including acute kidney injury (AKI). AKI is generally a consequence of tubular obstruction by luminal aggregates of LC, a pattern known as "cast nephropathy". Monoclonal Ig LC can also be found as intracellular crystals in glomerular podocytes or proximal tubular cells. Proliferative glom

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… Fibrillar or immunotactoid deposits can be observed also in lymphoproliferative disorders, such as B-cell lymphomas [ 78 , 84 ], while cryoglobulins span across membranoproliferative GN, mixed essential cryoglobulinemia, or systemic lupus erythemathosus (SLE) [ 86 , 87 ]. …

CAR T-клеточная терапия в раковых заболеваниях: учет перспектив ухода за больными в целях улучшения ухода за больными.

Цимерная антигенная рецепторная (CAR) T-клетотерапия представляет собой значительный прогресс в лечении рака, особенно в случае гематологовых магнестов. В настоящее время изучаются различные стратегии иммунотерапии рака, включая цитокины, вакцины от рака, ингибиторы иммунной системы, иммуномодуляторы моноклонных антител и т.д. Эта терапия продемонстрировала впечатляющую эффективность в лечении таких заболеваний, как острая лимфоболическая лейкемия (АЛЛ), диффузная большая лимфома B-клеток (DLBCL) и миелома, часто приводящая к полной ремиссии в рефрактных случаях. Вместе с тем клиническое приме

Роль питания и кишечного микробиома в развитии множественной миеломы и ее болезней-прекурсоров.

Множественная миелома (ММ) — вторая по распространенности гематологическая гелингология, характеризующаяся нерегулируемым моноклонным распространением в костном мозге. Моноклональная гаммопатия неопределённого значения (MMUS) и мрачная миелома (SMM) являются предмалярными условиями, которые могут развиваться до MM. Определение факторов этиологического риска для ММ и его болезней-прекурсоров имеет решающее значение для профилактики. Ожирение, питание, уровень витамина D и изменение кишечного микробиота были определены в качестве факторов образа жизни, влияющих на риск MM и MGUS. При возникновен

Multiple Myeloma Outpatient Transplant Program in the Era of Novel Agents: State-of-the-Art.

Multiple myeloma (MM) is the most common indication for autologous stem cell transplantation (ASCT), and outpatient models have been widely developed in this setting. Although numerous studies have demonstrated the safety and feasibility of outpatient ASCT, it is not a routine procedure. Stringent guidelines for patient selection and clinical management, including functional status, caregiver support, and psychological aspects, are essential to identify eligible patients. However, there is still no general agreement on these criteria. Quality of life data are limited and contradictory. There i

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… Aapro MS, Bohlius J, Cameron DA, Dal Lago L, Donnelly JP, Kearney N, et al. 2010 update of EORTC guidelines for the use of granulocyte-colony stimulating factor to reduce the incidence of chemotherapy-induced febrile neutropenia in adult patients with lymphoproliferative disorders and solid tumours. …