Turkish Journal of Hematology最新文献

Objective: Hemophilia A (HA) is a hereditary X-linked bleeding disorder secondary to deficiency of the clotting factor VIII (FVIII). Emicizumab is a monoclonal antibody that replaces the function of the activated FVIII and prevents bleeding in HA patients. Emicizumab is expected to ameliorate bleeding risk in those patients together with subsequent complications. However, there is a scarcity of data about its safety and efficacy in patients with HA. We aimed to evaluate the safety and efficacy of emicizumab prophylaxis in Egyptian pediatric patients with HA.

Materials and methods: A prospective cohort study was carried out with 88 HA patients who received emicizumab prophylaxis. Breakthrough bleeding episodes and the annualized bleeding rate (ABR) were reported for all patients before and after emicizumab prophylaxis. Also, all adverse events during prophylaxis were documented to evaluate the safety of emicizumab.

Results: Joint bleeds occurred in 94% of the patients. Among those patients, 58% had one target joint, 36.4% had more than one target joint, and 5.6% had no target joints. Furthermore, 17% of patients were positive for FVIII inhibitors. The median annualized joint bleeding rate was reduced remarkably after emicizumab prophylaxis (36 before versus 0 after emicizumab). The median ABR was 48 before emicizumab versus 0 after emicizumab. Eight patients experienced mild breakthrough bleeding episodes. The most common adverse events were local reactions at injection sites, headache, arthralgia, fever, and diarrhea.

Conclusion: Prophylaxis using emicizumab was associated with a significantly lower bleeding rate in HA patients with and without inhibitors. The majority of patients had zero bleeds with emicizumab prophylaxis.

Objective: B-HOLISTIC was a real-world retrospective study of treatment patterns and clinical outcomes in Hodgkin lymphoma (HL) in regions outside Europe and North America. This subgroup analysis reports findings from Saudi Arabia, Türkiye, and South Africa.

Materials and methods: Patients aged ≥18 years and diagnosed with stage IIB-IV classical HL receiving frontline chemotherapy (frontline cHL) and/or with relapsed/refractory HL (RRHL) from January 2010 to December 2013 were assessed. The primary endpoint was progression-free survival (PFS) in patients with RRHL.

Results: Overall, 694 patients (RRHL: n=178; frontline cHL: n=653) were enrolled. Among patients with RRHL, >80% received first salvage chemotherapy. The most common first salvage regimens were etoposide, methylprednisolone, cytarabine, and cisplatin in Saudi Arabia (78.3%) and dexamethasone, cytarabine, and cisplatin in Türkiye (36.1%) and South Africa (40%). Median PFS (95% confidence interval [CI]) in the RRHL group was 5.1 (3.0-15.9), 19.7 (7.5-not reached), and 5.2 (1.1-10.1) months in Saudi Arabia, Türkiye, and South Africa, respectively. The 5-year PFS and overall survival (95% CI) rates in patients with RRHL were 33.2% (21.6-45.2) and 78.2% (65.9-86.5) in Saudi Arabia, 42.5% (29.5-54.9) and 79.4% (67.2-87.5) in Türkiye, and 13.1% (4.2-27.0) and 53% (35.5-67.8) in South Africa, respectively.

Conclusion: This study showed that the clinical outcomes in Türkiye and Saudi Arabia were generally comparable with those of Western countries during the study period, although Saudi Arabia had lower PFS rates. Conversely, the clinical outcomes in South Africa were suboptimal, emphasizing the need for novel therapies and improved progression to stem cell transplantation. These data may serve as a control group for future studies in these countries and inform clinical decision-making.

Objective: Despite advancements in treatment, multiple myeloma (MM) remains a challenging hematologic malignancy. It is crucial to stratify risk and perform prognostic assessment with various markers, including the expression of cluster of differentiation 56 (CD56) and cluster of differentiation 117 (CD117). However, the relationship of these markers with MM-related survival remains unclear. In this context, the objective of this study was to investigate the prognostic implications of CD56 and CD117 expression and associated clinical features in MM patients.

Materials and methods: The population of this retrospective single-center study consisted of adult MM patients whose CD56 and CD117 expression levels were analyzed. Patients were divided into four groups according to their immunophenotypes: CD56⁺CD117^-, CD56^-CD117⁺, CD56⁺CD117⁺, and CD56^-CD117^-. These groups were compared in terms of demographic and clinical characteristics, response to treatment, and survival outcomes.

Results: Of the 168 MM patients included in the study, CD56 positivity, CD117 positivity, CD56 and CD117 double positivity, and CD56 and CD117 double negativity were observed in 57.1%, 38.1%, 21.4%, and 26.2%, respectively. Patients with double positivity had significantly higher cytogenetic risk and significantly lower overall response rate (ORR) compared to other patients (p<0.001 for both). ORR and overall survival (OS) were significantly lower in CD56-positive patients than in CD56-negative patients (p=0.017 and p=0.004, respectively). Mortality rates were significantly higher in CD56-positive and CD117-positive patients than in double-negative patients (p<0.001 and p=0.002, respectively). Double-negative patients had significantly lower ORR and OS and higher mortality than others (p=0.001, p=0.002, and p<0.001, respectively). High cytogenetic risk was found to be an independent predictor of shorter OS (p>0.001).

Conclusion: This study’s findings revealed that MM patients with CD56 and CD117 double positivity had poorer prognosis, lower ORR, shorter OS, and higher mortality.

Objective: We investigated the occurrence and characteristics of secondary solid cancers (SSCs) in patients with Philadelphia chromosome-negative myeloproliferative neoplasms (Ph- MPNs) from Türkiye. We identified the potential risk factors for SSC development, including the impact of cytoreductive therapies, and we assessed the influence of SSC on patient survival.

Materials and methods: A total of 1013 Ph- MPN patients diagnosed between 1995 and 2022 were retrospectively analyzed. Data related to demographics, clinical and laboratory parameters, SSC development, cytoreductive therapy exposure, and survival outcomes were collected. Statistical analyses were performed using IBM SPSS Statistics 26.0.

Results: Of the analyzed Ph- MPN patients, 6.6% developed SSC, with carcinoma being the most common type. Older age at the time of Ph- MPN diagnosis and male sex were associated with SSC occurrence. Ph- MPN patients diagnosed with SSC and patients with no diagnosis of SSC showed no significant difference in complete blood count results, spleen size, Ph- MPN diagnostic groups, or driver mutation frequencies. However, patients with SSC had a higher frequency of arterial thrombosis and a tendency towards an increased rate of total thrombosis (p=0.030 and p=0.069, respectively). In multivariate analysis, arterial thrombosis was the sole independent risk factor and interferon (IFN)-based therapy was the sole protective factor for SSC development. Median overall survival (OS) did not differ between patients with and without SSC except for polycythemia vera patients with SSC, who had shorter OS (175±15 versus 321±26 months, respectively; p=0.005).

Conclusion: This study highlights the prevalence and characteristics of SSCs in Turkish patients diagnosed with Ph- MPNs. Arterial thrombosis was associated with increased SSC risk while IFN-based therapy offered potential protection from SSC. Screening for SSC in Ph- MPN patients with arterial thrombosis may be valuable. These findings emphasize the importance of malignancy screening in Ph- MPN patients, especially in high-risk subgroups, and call for further research to elucidate the underlying mechanisms and optimize treatment strategies.

Background: Fanconi anemia is the most common inherited bone marrow failure syndrome. HSCT remains the only curative treatment for hematological manifestations of FA. Despite restoration of long-term hematopoiesis, patients continue to remain at risk of late effects.

Objectives: In our study, we aimed to reveal the problems that occur in the long-term follow-up of FA patients, and point out an ongoing need for the improvement of long-term follow-up guidelines for childhood transplant survivors with FA.

Study design: In this single centered, cross-sectional study, we analyzed the long-term outcome of 36 patients with FA according to current recommendations with a median age of 18.1 years (range: 6.1-36 years, male/female, 24/12) who underwent a HSCT at Pediatric Bone Marrow Transplantation (BMT) Unit between 1995 and 2019 and survived at least one year post-transplant.

Results: The median long-term follow-up time was 8 years (range, 1-25 years). Gonadal dysfunction was detected in about 35% of our patients. 31% of the patients had hypergonadotropic hypogonadism, 4 % had hypogonadotropic hypogonadism. When the patients were evaluated for growth impairment, 7 of 12 patients who reached their final adult height and 12 of 21 patients who didn't complete their growth, had height standard deviation score below -2 SD. Three patients (9%) developed subclinical hypothyroidism, two (6%) had overt hypothyroidism and one (3%) had central hypothyroidism. Although, none of our patients fully met the criteria for metabolic syndrome, 23% of the patients had insulin resistance and 39% had dyslipidemia. Evaluation of organ dysfunctions revealed that almost 50% of the patients had obstructive and 21 % had restrictive changes in their pulmonary function tests. Hepatosteatosis was detected in 15% of the patients and mild valve dysfunction was detected in 50 % of evaluable patients. Three patients developed secondary malignancies. Squamous cell cancer developed in 2 patients and basal cell cancer in one patient.

Conclusion: A risk-defined multidisciplinary approach for long-term follow up of children with FA undergoing HSCT is essential for early detection and management of late effects.