Multi-refractory immune thrombocytopenia (ITP) is not uncommon and associated with high morbidity and mortality rates. Although the precise mechanism of ITP is not yet fully understood, a therapeutic approach that relies on using a single agent in each treatment line may not be sufficient in this population. We report the case of a 67-year-old female with long-standing multi-refractory ITP treated with a combination of Daratumumab and Romiplostim who achieved a durable response for more than 42 weeks. Owing to the presentation of chronic and refractory disease, we used a dual-agent approach to address early immune destruction and promote megakaryocyte platelet production. Three doses of Daratumumab were administered during the induction phase (weeks 0,1,5) and then at less frequent intervals - every 4– 12 weeks for total of 4 doses during the maintenance phase. Romiplostim was administered weekly, with dose modification depending on the platelet count. We hypothesize that when combined with thrombopoietin receptor agonists (TPO-RAs), daratumumab administered at less frequent intervals over an extended period can be safely used, resulting in a prolonged response.

None Received: 07 April 2024 Accepted after revision: 15 June 2024 Accepted Manuscript online: 18 June 2024 © . Thieme. All rights reserved. Georg Thieme Verlag KG Rüdigerstraße 14, 70469 Stuttgart, Germany

Background Measurement of factor VII (FVII) activity does not enable prediction of bleeding tendency in individuals with inherited FVII deficiency. Objective To characterize the molecular and functional features of FVII in a family with FVII deficiency and correlate them with the bleeding tendency. Patients/Methods We studied 7 family members with very low FVII activity using prothrombin time (PT), activated factor VII (FVIIa), FVII activity level, and thrombin generation. The factor 7 gene was sequenced and the mutation was analyzed by prediction software. Results The proband has very low FVII activity (0%–4%), with PT ranging between 5% to 18% depending on the tissue factor (TF) origin. Direct sequencing demonstrated a single homozygous nucleotide substitution G > A in exon 6, predicting a novel missense mutation Cys164Tyr. Three members of the family were found to be heterozygous carriers of this mutation. One of them was a compound heterozygote, carrying both the Cys164Tyr and Ala244Val mutation (linked to Arg353Gln polymorphism). Her FVII activity and antigen levels were 3%–7% and 8%, respectively. The other heterozygous carriers demonstrated FVII activity of 41%–54%, FVII antigen of 46%–66%, and FVIIa activity of 30%. FVIIa was undetectable in the homozygous and compound heterozygous subjects. Thrombin generation was normal in the presence of calcium, but no response to TF addition was observed in the homozygous proband, and a reduced response was observed in the compound heterozygous subject. Conclusion The patient homozygous for the “Carmel” mutation has mild clinical manifestations despite very low FVII activity, which correlates with thrombin generation results.

Multiple Myeloma (MM) is characterized by multifaceted symptoms stemming from the disease itself, treatment complications, and involvement of various organs. MM significantly impacts the lives of patients and caregivers. This study aims to assess Patient Reported Outcome Measures (PROMs) and Family Reported Outcome Measures (FROMs) alongside conventional evaluation tools. A quantitative, prospective, observational study was conducted involving active MM patients. Participants completed PROMs/FROM-16 questionnaires quarterly for 12 months. Spearman correlation analysis was employed to explore associations between PROMs/FROM-16 and disease evaluation parameters. Friedman’s test and Wilcoxon’s test compared mean PROMs/FROM-16 scores at study onset with the following months. A better disease evaluation correlated with improvements in reported disease symptoms or treatment side effects by patients and with a reduced burden reported by caregivers (FROM-16). Additionally, favorable results in calcium levels, renal insufficiency, anemia, and bone lesions (CRAB) were associated with higher PROMs scores and less impact on caregivers. Moreover, patient PROMs scores reflected caregiver challenges and their ability to cope with the patient’s illness. Most of the domains in PROMs demonstrated improvement, especially- quality of life, disease symptoms, body image scale, and future perspective, which improved 6 points that were defined in previous studies as minimal important difference. Finally, the results of our study showed that the total FROM-16 score improved from a mean score of 14.95 to 10.86 between the third month of the study, compared to the sixth month of the study, i.e., higher than 4, the minimal important change as defined in a previous study. This study underscores the utility of PROMs/FROM-16 in evaluating patient status from their perspective, facilitating tailored treatment approaches. It emphasizes the necessity of further research into the caregiver’s experience with MM.

Background: Patients with severe chronic anemia often require frequent blood transfusions. Many are elderly with comorbidities and limited mobility, making regular hospital visits burdensome. In some cases, patients may receive transfusions despite hemoglobin levels being above the clinical threshold due to logistical challenges, leading to unnecessary exposure to risks, inefficient use of blood units, and resource strain. This study aims to evaluate the use of point-of-care (POC) hemoglobin measurements under controlled outpatient clinic conditions, as an initial step toward potential future home-based monitoring by the patients or their caregivers, with the goal of optimizing transfusion timing, aiming to reduce unnecessary hospital visits while maintaining patient safety. Methods: A total of 127 patients from a hemato-oncology outpatient clinic at Carmel Medical Center were evaluated using a nurse-operated POC device to sample capillary blood, with 236 paired measurements concurrently analyzed via venous blood in the laboratory. Demographic and clinical data were assessed to evaluate factors associated with agreement between POC and laboratory measurements. Statistical analysis included Bland–Altman plots and Pearson correlation coefficients. Results: The POC device showed a moderate correlation with laboratory results (r = 0.73, p < 0.001), with a mean difference of 1.20 g/dL (SD = 1.94 g/dL) but wide limits of agreement (−3.20 to 5.50 g/dL). No significant differences were observed across demographic or clinical subgroups. Notably, all 156 paired measurements with POC-measured hemoglobin >7 g/dL were confirmed by laboratory testing. Conclusions: Although POC hemoglobin devices are not suitable as standalone tools for routine monitoring of chronic anemia, the high negative predictive value observed at the 7 g/dL threshold suggests that they may be useful for ruling out severe anemia. If validated in larger multicenter and home-use studies, POC Hb devices might contribute to reducing unnecessary hospital visits and transfusions.