Clinician's knowledge and awareness of patient-reported outcomes in immune thrombocytopenia: Results of a national survey (ITP NET, GIMEMA Working Group Anemia and Thrombocytopenia).

Background: Patient-reported outcomes (PROs) are increasingly recognized in hematology, yet clinician awareness remains limited. In immune thrombocytopenia (ITP), an autoimmune disease, symptoms like fatigue impact quality of life (QoL).

Design And Methods: This Italian survey evaluated hematologists' knowledge of PROs within the Italian ITP NET network.

Characterization of Breakthrough Hemolysis in Patients With Paroxysmal Nocturnal Hemoglobinuria: An International Multicenter Experience.

Breakthrough hemolysis (BTH) is defined as a hemolytic exacerbation in a patient with paroxysmal nocturnal hemoglobinuria (PNH) treated with complement inhibitors (CIs). In the current era of several terminal and proximal inhibitors, there are no guidelines for defining BTH and its severity, and clinical management is not standardized. This retrospective, observational, and multicentric study evaluated BTH frequency and severity in PNH patients treated with complement inhibitors from 2007 until February 2025 at 10 centers across Italy (9 centers) and the United Kingdom (1 center).

Unmet Needs and Their Impact on Quality of Life and Symptoms in Myelodysplastic Neoplasm Patients and Caregivers.

Background/objectives: The aim of this study was to assess the unmet needs of myelodysplastic neoplasm (MDS) patients and their caregivers, focusing on how these needs impact quality of life (QoL) and daily functioning. MDS predominantly affects older adults. It is often complicated by severe red blood cell transfusion-dependent anemia and may require frequent hospital visits, conferring a substantial burden on patients and caregivers.

Psychometric properties and meaningful change thresholds for the QOL-E instrument in patients with myelodysplastic neoplasms.

Background: Myelodysplastic neoplasms (MDS) are characterized by ineffective hematopoiesis, peripheral blood cytopenias, and an increased risk of progression to acute myeloid leukemia. One of the main treatment goals is improving quality of life (QoL), particularly for patients with lower-risk MDS (LR-MDS) who may live longer with compromised QoL. The QOL-E is a patient-reported outcome (PRO) measure specifically developed to address the lack of a health-related QoL questionnaire for patients with MDS.

Real-world evidence of pegcetacoplan in patients with paroxysmal nocturnal haemoglobinuria: A nationwide Italian study.

In this study, we collected real-world evidence on the use of pegcetacoplan among 22 Italian patients with paroxysmal nocturnal haemoglobinuria showing suboptimal response to anti-C5 treatments eculizumab and ravulizumab. Most patients exhibited a complete or good response as per the criteria of the European Bone Marrow Transplant group (Risitano et al. Front Immunol 2019) and median haemoglobin improvement from baseline was +3.

Long-term Sustained Response to Lenalidomide after Clearance of Bone Marrow Blasts by Azacytidine in High-risk Myelodysplastic Syndromes with Del5q: A Case Report.

Introduction: This report discusses a rare case involving a patient with high-risk (HR) Del(5q) myelodysplastic syndrome (MDS) who achieved a long-term response to lenalidomide after having received six cycles of azacytidine. The latter treatment led to the clearance of blast cells from the bone marrow (BM).

Case Representation: As per current clinical practice, patients with HR MDS receive azacytidine continuously until the disease progresses or the occurrence of unmanageable side effects.

Translational Research on Azacitidine Post-Remission Therapy of Acute Myeloid Leukemia in Elderly Patients (QOL-ONE Trans-2).

The achievement of complete remission (CR) is crucial for acute myeloid leukemia (AML) patients undertaking curative therapy, but relapse often occurs within months, highlighting the need for strategies to prolong disease-free survival (DFS). Our phase III study compared the efficacy and safety of azacitidine (AZA) to best supportive care (BSC) in elderly AML patients who achieved CR following intensive induction and consolidation therapy. This ancillary study (QOL-ONE Trans-2) evaluated biological changes in bone marrow using Next-Generation Sequencing (NGS).

Patient-reported outcomes in Hodgkin lymphoma trials: a systematic review.

Background: Lymphoma treatment can lead to long-term consequences such as fatigue, infertility and organ damage. In clinical trials, survival outcomes, clinical response and toxicity are extensively reported while the assessment of treatment on quality of life (QoL) and symptoms is often lacking.

Objective: We evaluated the use and frequency of patient-reported outcome (PRO) instruments used in randomized controlled trials (RCTs) for Hodgkin lymphoma (HL) and their consistency of reporting.

Guidelines for the Use and Reporting of Patient-Reported Outcomes in Multiple Myeloma Clinical Trials.

In the era of personalized medicine there is an increasing need for the assessment of patient-reported outcomes (PROs) to become a standard of patient care. Patient-reported outcome measures (PROM) are important in assessing significant and meaningful changes as a result of an intervention based on a patient's own perspective. It is well established that active multiple myeloma (MM) can be characterized by a high burden of disease and treatment-related symptoms, with considerable worsening of quality of life (QoL).

Eltrombopag for Low-Risk Myelodysplastic Syndromes With Thrombocytopenia: Interim Results of a Phase II, Randomized, Placebo-Controlled Clinical Trial (EQOL-MDS).

Purpose: In myelodysplastic syndromes (MDS), severe thrombocytopenia is associated with poor prognosis. This multicenter trial presents the second-part long-term efficacy and safety results of eltrombopag in patients with low-risk MDS and severe thrombocytopenia.

Methods: In this single-blind, randomized, placebo-controlled, phase-II trial of adult patients with International Prognostic Scoring System low- or intermediate-1-risk MDS, patients with a stable platelet (PLT) count (<30 × 10/mm) received eltrombopag or placebo until disease progression.

Azacitidine Post-Remission Therapy for Elderly Patients with AML: A Randomized Phase-3 Trial (QoLESS AZA-AMLE).

This phase-3 randomized multicenter trial evaluated the efficacy of subcutaneous azacitidine (AZA) post-remission therapy vs. best supportive care (BSC) in elderly acute myeloid leukemia (AML) patients. The primary endpoint was the difference in disease-free survival (DFS) from complete remission (CR) to relapse/death.

Does Etelcalcetide reverse myelofibrotic bone changes due to hyperparathyroidism? A case report.

Secondary hyperparathyroidism (SHPT) in dialysis is common. A young man on chronic hemodialysis with SHPT developed pancytopenia with resistant anemia requiring transfusions. A bone marrow biopsy showed grade 3 fibrosis, depleted cellularity, osteosclerosis, and decreased myelopoiesis.

The Reporting, Use, and Validity of Patient-Reported Outcomes in Multiple Myeloma in Clinical Trials: A Systematic Literature Review.

Background: Patient-reported outcomes (PROs) are becoming increasingly important in supporting clinical outcomes in clinical trials. In multiple myeloma (MM), PRO measurement is useful to reveal how treatment affects physical, psychosocial, and functional behaviour as well as symptoms and treatment-related adverse events to evaluate the benefit-risk ratio of a particular drug or drug combination. We report the types of PRO instruments used in MM, the frequency in which they are utilised in randomised controlled trials (RCTs), and the consistency of their reporting.

Relationship between Serum Ferritin and Outcomes in β-Thalassemia: A Systematic Literature Review.

Among the difficulties of living with β-thalassemia, patients frequently require blood transfusions and experience iron overload. As serum ferritin (SF) provides an indication of potential iron overload, we conducted a systematic literature review (SLR) to assess whether SF levels are associated with clinical and economic burden and patient-reported outcomes (PROs). The SLR was conducted on 23 April 2020 and followed by analysis of the literature.

Managing Fatigue in Patients with Paroxysmal Nocturnal Hemoglobinuria: A Patient-Focused Perspective.

The most frequently reported symptom in patients with paroxysmal nocturnal hemoglobinuria (PNH), a disease characterized by complement mediated hemolysis and chronic anemia, is "fatigue". The latter seems the best word to communicate patient' perception of personal health status and disease impact on daily living, namely quality of life (QoL). Objectivating QoL and grading patient's fatigue is one of the most difficult medical tasks given the highly heterogeneous communication skills of patients and caregivers and the multitude of meanings that might be attributed to this term.

A Systematic Literature Review of the Relationship between Serum Ferritin and Outcomes in Myelodysplastic Syndromes.

Anemia is the most common form of cytopenia in patients with myelodysplastic syndromes (MDS), who require chronic red blood cell transfusions and may present high serum ferritin (SF) levels as a result of iron overload. To better understand the potential effects of high SF levels, we conducted a systematic literature review (SLR) to identify evidence on the relationship between SF levels and clinical, economic, or humanistic outcomes in adult patients with MDS. Of 267 references identified, 21 were included.

Health-Related Quality of Life Outcomes in Patients with Myelodysplastic Syndromes with Ring Sideroblasts Treated with Luspatercept in the MEDALIST Phase 3 Trial.

Patients with myelodysplastic syndromes (MDS) often experience chronic anemia and long-term red blood cell transfusion dependence associated with significant burden on clinical and health-related quality of life (HRQoL) outcomes. In the MEDALIST trial (NCT02631070), luspatercept significantly reduced transfusion burden in patients with lower-risk MDS who had ring sideroblasts and were refractory to, intolerant to, or ineligible for prior treatment with erythropoiesis-stimulating agents. We evaluated the effect of luspatercept on HRQoL in patients enrolled in MEDALIST using the EORTC QLQ-C30 and the QOL-E questionnaire.