Therapeutic adherence and assessment of satisfaction patients with multiple myeloma treated with immunomodulatory drugs in a "real-world" study: Experiences of the Polish Myeloma Group.

INTRODUCTION: Therapeutic adherence (TA) is one of the most important factors influencing the effectiveness of treatment. Oral anti-cancer drugs are increasingly used to treat malignancy including multiple myeloma (MM). Our study aimed to determine TA of patients with MM treated with IMiDs, to identify TA risk factors, and to determine satisfaction with medical care during the treatment with IMiDs. METHODS: A cross-sectional survey-based study involving adult patients with MM treated with IMiDs. RESULTS: Between January 2021 and May 2021, 267 patients with MM were enrolled in the study. The dosing schedule was declared as easy by 71.8% of patients, as standard for 24.0%, and difficult for 4.2% of patients. During MM treatment, 85.0% of patients did not skip any IMiDs dose, and 87.6% did not skip the IMiDs dose in the last cycle of chemotherapy. Identified factors affecting TA included the treatment duration and education level. In addition, depending on the patient's well-being, gender, and household companionship influenced TA. Satisfaction with medical care during the treatment with IMiDs was declared by 95.5% of patients with MM. In our cohort, 95.5% of patients were satisfied with the information they received from the hematologist during treatment with IMiDs. CONCLUSIONS: Patients with MM treated with IMiDs are highly adherent to treatment. With time from the beginning of treatment, patients need more attention and motivation to adhere to the therapy rules.

Demographic, Hematologic, and Clinical Features of Myelodysplastic Syndrome Patients: Results from the First Polish Myelodysplastic Syndrome Registry.

Epidemiological studies on myelodysplastic syndromes (MDS) in Middle-Eastern Europe are scarce. No data about the demographic, clinical, and laboratory features of Polish MDS patients have been published. The aim of this study was to assess the epidemiological data and toxic exposure of Polish MDS patients and their association with hematological parameters and clinical outcomes. For 15 months, 966 living MDS patients were enrolled at 24 centers (12 university and 12 community hospitals). Follow-up was conducted for the next 55 months. The percentage of patients older than 80 years (16%) was between the values for Eastern and Western countries. In patients younger than 55 years, a female predominance was observed (male/female ratio 0.70:1 vs. 1.29:1; p < 0.001). Female patients had higher platelet counts (160 × 109/l vs. 111 × 109/l; p < 0.001). Patients exposed to chemicals were younger than patients without such exposure; their median age at MDS diagnosis was 66 vs. 70 years (p = 0.037). Smokers had significantly lower hemoglobin concentrations (8.6 vs. 9.1 g/dl; p = 0.032) and lower platelet counts (99 × 109/l vs. 137 × 109/l; p < 0.001) than nonsmokers. We provide the first description of the characteristics of Polish MDS patients. Females predominated in the group aged <60 years and they had higher platelet counts. The course of the disease is affected by toxic exposure and smoking.

Prognostic factors and clinical characteristics of patients with newly diagnosed non-secretory multiple myeloma in the era of new drugs in "real-world" study: Experiences of the Polish Myeloma Group.

BACKGROUND: Non-secretory multiple myeloma (NSMM) accounts for approx. 2-3% of multiple myeloma (MM) cases. Due to the rare occurrence and ineligibility of patients with NSMM to participate in clinical trials, we have limited data on treatment efficacy and the clinical course in these patients. Most of the literature consists of case reports and small retrospective studies. OBJECTIVES: The study aimed to analyze patient characteristics, prognostic factors and treatment outcomes in newly diagnosed (ND) NSMM. MATERIAL AND METHODS: This is a multicenter, retrospective analysis of 43 patients with NSMM diagnosed between June 2010 and September 2021, conducted in 8 Polish hematology centers. RESULTS: The median overall survival (OS) was 103 months (95% confidence interval (95% CI): 20-72). The most common cause of death was MM disease progression. The overall response rate (ORR) was 84.6%; complete response (CR), very good partial response (VGPR), partial response (PR), and no response (NR) rates were 20.5%, 46.2%, 17.9%, and 15.4%, respectively. In multivariable analysis, factors contributing to worse OS included International Staging System stage 3 (ISS-3) (p = 0.0277), anemia (Hb <10 g/dL or >2 below upper limit of normal value (ULN), p = 0.0270), renal insufficiency (RI, serum creatinine >2 mg/dL, p = 0.0476), and serum albumin <5.5 mg/L (0.0408). CONCLUSIONS: Non-secretory multiple myeloma is a rare subtype of MM. This small study demonstrates that outcomes are comparable to secretory MM. However, the inclusion of this subset of patients in clinical trials is essential to assess prognosis, treatment efficacy and clinical outcomes.

Immune thrombocytopenia in patients with chronic lymphocytic leukemia treated with cladribine-based regiments or chlorambucil--follow-up of PALG-CLL randomized trials.

OBJECTIVES: The relationship between treatments of chronic lymphocytic leukemia (CLL) with cladribine (2-CdA) or chlorambucil and immune thrombocytopenia (IT) has not been yet determined. METHODS: The records of 777 patients in two randomized Polish Adult Leukemia Group (PALG)-CLL programs treated with these agents were retrospectively analyzed. RESULTS: Immune thrombocytopenia occurred in 55 of 777 (7.1%) patients. No significant differences in IT prevalence were seen between patients on chlorambucil or 2-CdA-based regiments (P = 0.33). IT developed at a median time of 0.499 yr (0.06-4.8) from the start of CLL therapy. This time was significantly longer in patients treated with chlorambucil (2.03 yr, 95% CI: 0.06-4.22) in relation to patients treated with 2-CdA-based regiments (0.52 yr, 95%CI: 0.34-0.69, P = 0.049). Overall survival (OS) of patients with IT and those without IT were similar (2.65 yr vs. 3.2 yr P = 0.23) but the severity of bleeding was more pronounced in the 2-CdA group. The responses to IT therapy were 35%, 54% and 75% for steroids, chemotherapy and splenectomy, respectively. CONCLUSIONS: In this study, an unexpectedly high percentage of IT incidence was demonstrated in patients with CLL requiring chemotherapy. Although no marked differences were seen in IT frequency in patients treated with 2-CdA-based regiments compared to chlorambucil regimen, the clinical course of hemorrhagic diathesis was more severe in 2-CdA group. Also, the time elapsed from study screening to IT diagnosis was significantly shorter in the 2-CdA group than in the chlorambucil group suggesting a causative relationship. The appearance of IT did not influence the median time of OS.

[Immunomodulatory drugs in the treatment of primary systemic light chain amyloidosis]. / Leki immunomodulujace w leczeniu pierwotnej ukladowej amyloidozy lancuchów lekkich.

Primary systemic immunoglobulin light chain amyloidosis (AL.) is an incurable clonal plasma cell disorder in which fragments of Ig light chain are deposited in tissues. High dose melphalan and hematopoietic cell transplantation (SCT) is a preferred technique, but only 20% of patients are eligible. Nontransplant candidates can be offered MelDex (melphalan-dexamethasone). Demonstrate comparable efficacy of treatment protocols including immunomodulatory drugs such as TCD (thalidomide, cyclophosphamide, dexamethasone), LMP (lenalidomide, melphalan, prednisone), or bortezomib in combination with dexamethasone. Results of treatment of patients with AL. based on immunomodulatory drugs are promising but require further multicenter clinical trial comparing the MelDex. The main obstacle to effective treatment of AL. still remains a late diagnosis of the disease.

Low serum albumin level deteriorates prognosis in azacitidine-treated myelodysplastic syndromes patients - results of the PALG study 'PolAZA'.

BACKGROUND: Azacitidine (AZA) is the standard of care for higher-risk myelodysplastic syndrome (HR-MDS) patients ineligible for intensive therapy. Clinical outcome discrepancies reported in clinical trials and real-life settings stimulate the search for new prognostic factors. METHODS: We retrospectively evaluated 315 MDS, 20-30% blast acute myeloid leukemia (AML) and chronic myelomonocytic leukemia (CMML) patients treated with azacitidine in 12 centers cooperating within the Polish Adult Leukemia Group (PALG). RESULTS: The median number of AZA cycles was 7 (1-69) and 24% patients received fewer than 4 cycles (early failure, EF). Serum albumin level was an independent predictor of EF occurrence. Complete remission (CR) was obtained in 20% and partial remission (PR) in 12% of patients. Hematologic improvement - erythroid (HI-E), neutrophil (HI-N), or platelet (HI-P) was achieved in 51%, 36%, and 48% of patients, respectively. No factors significantly predicted CR or PR in the multivariate analysis. For HI-E and HI-P, lower LDH level predicted response. Median survival was 15 (13-19) months. Lower serum albumin level, serious infection and receiving <4 AZA cycles independently predicted a worse overall survival (OS) (p < 0.05). CONCLUSION: Serum albumin assessment before azacitidine treatment can help to identify patients with higher risk of early failure and worse clinical outcome.

Cladribine in a weekly versus daily schedule for untreated active hairy cell leukemia: final report from the Polish Adult Leukemia Group (PALG) of a prospective, randomized, multicenter trial.

Cladribine (2-chlorodeoxyadenosine, 2-CdA) treatment-associated infections may shorten potentially long-term survival in hairy cell leukemia (HCL). In search of the optimal mode of 2-CdA administration, 132 patients with untreated HCL were randomized to receive either standard 5-day 2-CdA protocol or a novel schedule of 6 weekly 2-CdA infusions suggested to be less toxic. Analysis of treatment response confirmed similar complete remission rates, overall response rates, progression-free survival, and overall survival in both 2-CdA protocols. However, we did not observe lower toxicity in the weekly schedule. Of special interest, no significant differences were found in the rate of grade 3/4 infections (18% for daily and 26% for weekly protocol, difference -8.2%; 95% confidence interval [CI] -23.2% to 6.9%; P = .28) and the rate of septic deaths (3% for daily and 2% for weekly protocol, difference 1.4%; 95% CI -4.3% to 7.0%; P = .64). In conclusion, HCL treatment with weekly 2-CdA infusions is equally effective but no safer than the standard 5-day 2-CdA protocol.

[Study of subpopulations of lymphocytes in the bone marrow of patients with myelodysplastic syndromes]. / Badanie subpopulacji limfocytów w szpiku kostnym u chorych na zespoly mielodysplastyczne.

UNLABELLED: Myelodysplastic syndromes are heterogeneous group of clonal blood disorders. Various abnormalities in the immunoregulation system have been reported in patients with MDS. Most of studies were about lymphocytes in peripheral blood. There weren't reports described lymphopoesis in the peripheral blood after reclassification of MDS according WHO. We analyzed retrospectively subopulations of lymphocytes in bone marrow of patients with MDS recognized according to the WHO classification. INVESTIGATED GROUP: There were 58 patients, 24 women and 34 men. We recognized following subtypes of MDS: 5 pts with RA (8.5%), 22-RCMD (38%), 4-RCMD-RS (7%), 14-RAEB1 (24%), 9-RAEB2 (15.5%), 3-MDS-U (5%),1-5q-. METHODS: Cell staining was performed in whole bone marrow during routine phenotyping with using following antibodies: anti-CD3, anti-CD4, anti-CD8, anti-CD16, anti-CD56, anti-CD19. All of the results have been statistically tested by using t-Student test and Spearmans correlation. RESULTS: There percentage of cells CD3+ was statistically larger, lymphocytes CD19+ statistically smaller, than in healthy in pts with MDS. In analyzed subtypes of MDS: RCMD, RAEB 1 and RAEB2 we detected statistically larger percentage of cells: CD3+, CD3+CD4+, CD3+CD8+ and smaller lymphocytes CD 19+ percentage-than in normal BM. Statistically larger percentage of cells CD3-CD16+CD56+ in subtype RAEB2 than RAEB1 was only one statistically difference between subtypes MDS: RCMD, RAEB1 and RAEB2. Following dependencies were found out in correlation's study: negative correlation between percentage of whole lymphocytes and celullarity of BM, percentage of lymphocytes, CD3+ cells, CD3+CD4+ cells and quantative of granulopoesis; positive correlation between percentage of CD3-CD16+CD56+ cells and changes in intensification of granulopoesis and percentage of CD3+CD8+ and quantitative of dyserythropoesis and changes in intensification megakariopoesis. SUMMARY: Our studies show differences in percentage of whole lymphocytes in whole population of pts with MDS and supopulations: RCMD, RAEB1 and RAEB2 in comparison to healthy donors. We showed relationship between: percentage of lymphocytes CD3+ and changes of intensification granulopoesis, CD3+CD8+ cells and dyserythropoesis and megakariopoesis. Detected differences between subtypes RAEB1 and RAEB2 in precemtage cells CD3CD16+CD56+ verified changes between these 2 new isolated by WHO subtypes of MDS.