Myelodysplastic syndromes: Aspects of current medical care and economic considerations in Germany

Myelodysplastic syndromes (MDS) are a heterogeneous group of diseases mainly affecting older people. The use of an increasing number of therapeutic options depends on a systematic risk stratification of the patients. A high percentage of MDS patients need blood transfusions as supportive care, which influence quality of life and cause a great part of the costs generated by MDS therapy. In this article which is based on a workshop about the burden of MDS held in October 2006 in Munich, MDS is discussed with regard to different aspects: current therapies, transfusion medicine, geriatrics, quality of life, and health economic aspects

Clinical Impact of Pretransplant Multidrug-Resistant Gram-Negative Colonization in Autologous and Allogeneic Hematopoietic Stem Cell Transplantation

Abstract Multidrug-resistant Gram-negative bacteria (MDR-GNB) are an emerging cause of morbidity and mortality after hematopoietic stem cell transplantation (HSCT). Three-hundred forty-eight consecutive patients transplanted at our hospital from July 2012 to January 2016 were screened for a pretransplant MDR-GNB colonization and evaluated for clinical outcomes. A pretransplant MDR-GNB colonization was found in 16.9% of allo-HSCT and in 9.6% of auto-HSCT recipients. Both in auto- and in allo-HSCT, carriers of a MDR-GNB showed no significant differences in overall survival (OS), transplant-related mortality (TRM), or infection-related mortality (IRM) compared with noncarriers. OS at 2 years for carriers compared with noncarriers was 85% versus 81% ( P  = .262) in auto-HSCT and 50% versus 43% ( P  = .091) in allo-HSCT. TRM at 2 years was 14% versus 5% ( P  = .405) in auto-HSCT and 31% versus 25% ( P  = .301) in allo-HSCT. IRM at 2 years was 14% versus 2% ( P  = .142) in auto-HSCT and 23% versus 14% ( P  = .304) in allo-HSCT. In multivariate analysis, only grade III to IV acute graft-versus-host disease was an independent factor for reduced OS ( P P P P  = .207). We conclude that in this extended single-center experience, a pretransplant MDR-GNB colonization did not significantly influence OS, TRM, and IRM both in auto- and allo-HSCT settings and that MDR-GNB attributed mortality can be controlled in carriers when an early pre-emptive antimicrobial therapy is started in case of neutropenic fever

Frequency of Paroxysmal Nocturnal Hemoglobinuria Clone in Turkish Myelodysplastic Syndrome Group

Aim:Retrospective, cross-sectional, observational study to examine the frequency and features of paroxysmal nocturnal hemoglobinuria (PNH) clones in patients with myelodysplastic syndrome (MDS).Methods:Data were analyzed from the medical files of 41 MDS patients diagnosed and followed up in the hematology department at a referral center between 2006 and 2017. Descriptive data, cytogenetic and hematologic characteristics, prognostic features and PNH clone sizes were assessed. PNH clone sizes were evaluated using the fluorescently labeled inactive toxin aerolysin method.Results:The study population comprised 22 (53.7%) female and 19 (46.3%) male patients with confirmed MDS; the mean age of the patients was 68.20±9.84 years (range, 45-85). PNH clones were detected in eight (19.5%) patients. The number of patients with a PNH clone size of >10%, >1%, >0.1% and >0.01% was one, one, one and eigth, respectively (p<0.001 for all subgroups).Conclusion:These data indicate that PNH clones exist in approximately one-fifth of MDS patients. Further studies on a more extensive cohort are required to better understand the pathophysiological and clinical relationships between MDS and PNH

Role of CBL Mutations in Cancer and Non-Malignant Phenotype

CBL plays a key role in different cell pathways, mainly related to cancer onset and progres-sion, hematopoietic development and T cell receptor regulation. Somatic CBL mutations have been reported in a variety of malignancies, ranging from acute myeloid leukemia to lung cancer. Growing evidence have defined the clinical spectrum of germline CBL mutations configuring the so-called CBL syndrome; a cancer-predisposing condition that also includes multisystemic involvement char-acterized by variable phenotypic expression and expressivity. This review provides a comprehensive overview of the molecular mechanisms in which CBL exerts its function and describes the clinical manifestation of CBL mutations in humans

CPX-351 and allogeneic stem cell transplant for a therapy-related acute myeloid leukemia that developed after treatment of acute promyelocytic leukemia: a case report and review of the literature

Therapy-related myeloid neoplasms (t-MNs), which develop after cytotoxic, radiation, or immunosuppressive therapy for an unrelated disease, account for 7%–8% of acute myeloid leukemia (AML). Worse outcomes and consequently shortened survival are associated with t-MNs as compared with de novo AML. Therapy-related MNs are being reported with increasing frequency in successfully treated acute promyelocytic leukemia (APL), in particular, before the introduction of all-trans retinoic acid (ATRA) plus arsenic trioxide (ATO). Considering the high curability of APL, t-MNs represent one of the prognosis-limiting factors in this setting of leukemia. We report our experience with a patient who developed t-AML 15 years after treatment for APL. Treatment included three cycles of chemotherapy with CPX-351 (Vyxeos, Jazz Pharmaceuticals) followed, as in remission, by an allogeneic hematopoietic stem cell transplant. A review of available literature was also included