Browsing by Author "Kalwak, Krzysztof"
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Publication Characteristics, treatment and outcome of 15 to 18 years-old adolescents with Chronic Myeloid Leukemia (CML): The experience of the International Registry of Childhood CML (I-CML-Ped Study)(American Society of Hematology, 2021-11-23) Froment, Morgane; Suttorp, Meinolf; Ragot, Stephanie; Deutsch, Helene; Goyeau, Violaine; De Moerloose, Barbara; Brandalise, Silvia Regina; Borisevich, Marina; Sedlacek, Petr; Gunes, Adalet Meral; Lausen, Birgitte; Molines Honrubia, Antonio; Jakovljevic, Gordana; Versluys, Birgitta; Kalwak, Krzysztof; Hraskova, Andrea; Millot, Frederic; MERAL GÜNEŞ, ADALET; Uludağ Üniversitesi/Tıp Fakültesi; EXD-8400-2022Item Favourable outcome of de novo advanced phases of childhood chronic myeloid leukaemia(Elsevier, 2019-07) Millot, Frederic; Maledon, Natacha; Guilhot, Joelle; Kalwak, Krzysztof; Suttorp, Meinolf; Güneş, Adalet Meral; Bursa Uludağ Üniversitesi/Tıp Fakültesi/Dahili Tıp Bilimleri/Çocuk Sağlığı Ve Hastalıkları Bölümü.; 24072843300Background: Chronic myeloid leukaemia (CML) is very rare in children. The aim of the study is to report the experience within the I-CML-Ped study in children and adolescents presenting at diagnosis with advanced phase disease and to describe their characteristics and outcomes. Methods: Of 479 children and adolescents enrolled in the international registry for childhood chronic myeloid leukaemia (I-CML-Ped Study; www.clinicaltrials.gov NCT01281735), 36 children (7.5%) presented at initial diagnosis with CML in advanced phase according to the European Leukemia Net criteria. Results: Nineteen (4%) patients were diagnosed in accelerated phase (CML-AP), and among the 17 patients (3.5%) diagnosed in blastic phase (CML-BP), 70% presented with lymphoid immunophenotype. Initial treatment of CML-AP/CML-BP consisted of tyrosine kinase inhibitors (TKIs) with or without chemotherapy, leading to complete haematologic response in 33 of 36 (92%) patients. Seventeen patients proceeded to haematopoietic stem cell transplantation. At the last follow-up, 18 of 19 patients with de novo CML-AP are alive in at least major molecular response (MMR) (n = 16), in progression (n = 1) or in molecular relapse (n = 1) and 13 of 17 patients with de novo CML-BP are alive in at least MMR. Five-year overall survival rates are 94% (95% confidence interval [CI]: 66%-99%) and 74% (95% CI: 44%-89%) for patients diagnosed in CML-AP and CML-BP, respectively. Conclusion: Children with advanced phase at diagnosis of CML seem to have a better survival rate than that reported for advanced phases evolving under TKI treatment.Item Features and outcome of chronic myeloid leukemia at very young age: Data from the international pediatric chronic myeloid leukemia registry(Wiley, 2020-08-24) Millot, Fredic; Kalwak, Krzysztof; Lausen, Birgitte; Sedlacek, Petr; Versluys, A. Birgitta; Dworzak, Michael; De Moerloose, Barbara; Suttorp, Meinolf; Güneş, Adalet Meral; Bursa Uludağ Üniversitesi/Tıp Fakültesi/Çocuk Hematoloji Anabilim Dalı.; 0000-0002-0686-7129; JGX-6145-2023; 24072843300Introduction Chronic myeloid leukemia (CML) is rare in the first two decades of life comprising only 3% of newly diagnosed pediatric and adolescent leukemias. We studied the epidemiologic and clinical features of patients with CML diagnosed at younger than 3 years of age and evaluated treatment and long-term outcome. Method Data from the International Pediatric I-BFM/CML Registry were retrospectively analyzed using the European LeukemiaNet criteria of the year 2006. Characteristics and treatment outcome of patients Twenty-two patients (n = 22/479; 4.6%, male/female:14/8) were enrolled with a median age of 22 months (range, 10-34 m). Major symptoms comprised asthenia (30%), fever (30%), abdominal pain (20%), extramedullary signs (14%), hemorrhage (5%), and weight loss (5%). The extramedullary signs were specified in eight children: blueberry muffin (n = 1), sudden swollen abdomen (n = 1), sustained vomiting (n = 1), and cervical and inguinal lymph nodes (n = 5). Two of five children with cervical and inguinal lymph nodes were categorized as accelerated phase. Overall, 19 of 22 (86%) children were diagnosed in chronic phase, while the remaining three patients were in advanced phase. Median follow-up was 78 months (range, 7-196 m). Twenty-one out of 22 patients initially received imatinib, while one child received IFN + ARA-C. Imatinib was changed to second-line tyrosine kinase inhibitors (TKIs) in 29% of cases. During follow-up, 41% patients underwent stem cell transplantation (SCT). While on TKI, major molecular response (MMR) was achieved in 48% of children. Among the remaining patients, 21% are alive on TKI without MMR and 22% achieved complete molecular response following SCT. Twenty-one of 22 (95%) children are alive, while one patient died of posttransplant complications. Conclusion This report demonstrates for the first time the efficacy and long-term effects of upfront imatinib in the so far largest cohort of children with CML diagnosed at very young age.Item Generic formulations of imatinib for treatment of Philadelphia chromosome-positive leukemia in pediatric patients(Wiley, 2018-12) Suttorp, Meinolf; Metzler, Markus; Millot, Frederic; Shimada, Hiroyuki; Bansal, Deepak; Kalwak, Krzysztof; Sedlacek, Petr; Baruchel, Andre; Biondi, Andrea; Hijiya, Nobuko; Schultz, Kirk R.; Schrappe, Martin; Güneş, Adalet Meral; Uludağ Üniversitesi/Tıp Fakültesi/Çocuk Hematoloji Kliniği.; EXD-8400-2022; 24072843300Since the patent for imatinib has expired, the role of generic imatinib (GI) in the management of Philadelphia chromosome-positive (Ph+) leukemia in pediatric patients has had ongoing discussion. Some studies in adults demonstrated that equivalent doses of GI and branded imatinib (BI) result in comparable plasma concentrations and clinical efficacy. However, other studies found that GI users are more likely to stop imatinib, with intolerance and decreased persistence as the main causes. Economic factors also heavily influence GI selection. This article aims to review the present knowledge to support further discussion on the role of GI in the management of pediatric Ph+ leukemia.Publication Hematopoietic stem cell transplantation positively affects the natural history of cancer in nijmegen breakage syndrome(Amer Assoc Cancer Research, 2021-01-15) Wolska-Kusnierz, Beata; Pastorczak, Agata; Fendler, Wojciech; Wakulinska, Anna; Dembowska-Baginska, Bozena; Heropolitanska-Pliszka, Edyta; Piatosa, Barbara; Pietrucha, Barbara; Kalwak, Krzysztof; Ussowicz, Marek; Pieczonka, Anna; Drabko, Katarzyna; Lejman, Monika; Koltan, Sylwia; Gozdzik, Jolanta; Styczynski, Jan; Fedorova, Alina; Miakova, Natalia; Deripapa, Elena; Kostyuchenko, Larysa; Krenova, Zdenka; Hlavackova, Eva; Gennery, Andrew R.; Sykora, Karl-Walter; Ghosh, Sujal; Albert, Michael H.; Balashov, Dmitry; Eapen, Mary; Svec, Peter; Seidel, Markus G.; Tomaszewska, Agnieszka; Wiesik-Szewczyk, Ewa; Kreins, Alexandra; Greil, Johann; Buechner, Jochen; Lund, Bendik; Gregorek, Hanna; Chrzanowska, Krystyna; Mlynarski, Wojciech; Kilic, Sara S.; KILIÇ GÜLTEKİN, SARA ŞEBNEM; Bursa Uludağ Üniversitesi/Çocuk Sağlığı ve Hastalıkları Anabilim Dalı.; 0000-0003-4863-4443; 0000-0003-3089-6947; 0000-0002-5083-9168; 0000-0002-2561-0636; 0000-0001-5519-2730; 0000-0001-8949-047X; 0000-0002-9723-8351; 0000-0003-1174-5799; 0000-0001-5725-4835; 0000-0001-5922-4242; 0000-0002-7094-9129; 0000-0002-8760-0775; 0000-0002-3158-119X; 0000-0002-0262-1359; 0000-0001-9143-3263; 0000-0001-6590-5802; 0000-0001-6193-4243; 0000-0002-7647-2253; 0000-0003-0981-8661; 0000-0001-8571-2581; 0000-0001-8509-4453; 0000-0001-8748-5837; 0000-0001-5848-4501; 0000-0003-2714-5851; S-9959-2016; T-7487-2019; B-4557-2018; P-1827-2019; AAH-1658-2021; N-9951-2017; AAD-5720-2020; S-9592-2016Purpose: Nijmegen breakage syndrome (NBS) is a DNA repair disorder with a high predisposition to hematologic malignancies.Experimental Design: We describe the natural history of NBS, including cancer incidence, risk of death, and the potential effectiveness of hematopoietic stem cell transplantation (HSCT) in preventing both pathologies: malignancy and immunodeficiency.Results: Among 241 patients with NBS enrolled in the study from 11 countries, 151 (63.0%) patients were diagnosed with cancer. Incidence rates for primary and secondary cancer, tumor characteristics, and risk factors affecting overall survival (OS) were estimated. The cumulative cancer incidence was 40.21% +/- 3.5% and 77.78% +/- 3.4% at 10 years and 20 years of follow-up, respectively. Most of the tumors n = 95 (62.9%) were non-Hodgkin lymphomas. Overall, 20 (13.2%) secondary malignancies occurred at a median age of 18 (interquartile range, 13.7-21.5) years. The probability of 20-year overall survival (OS) for the whole cohort was 44.6% +/- 4.5%. Patients who developed cancer had a shorter 20-year OS than those without malignancy (29.6% vs. 86.2%; P < 10(-5)). A total of 49 patients with NBS underwent HSCT, including 14 patients transplanted before malignancy. Patients with NBS with diagnosed cancer who received HSCT had higher 20-year OS than those who did not (42.7% vs. 30.3%; P = 0.038, respectively). In the group of patients who underwent preemptive transplantation, only 1 patient developed cancer, which is 6.7 times lower as compared with nontransplanted patients [incidence rate ratio 0.149 (95% confidence interval, 0.138-0.162); P < 0.0001].Conclusions: There is a beneficial effect of HSCT on the long-term survival of patients with NBS transplanted in their first complete remission of cancer.Publication Nijmegen breakage syndrome: Clinical and immunological features, long-term outcome and treatment options - a retrospective analysis(Springer/Plenum Publishers, 2015-08-01) Wolska-Kusnierz, Beata; Gregorek, Hanna; Chrzanowska, Krystyna; Piatosa, Barbara; Pietrucha, Barbara; Heropolitanska-Pliszka, Edyta; Pac, Magorzata; Klaudel-Dreszler, Maja; Kostyuchenko, Larysa; Pasic, Srdjan; Marodi, Laszlo; Belohradsky, Bernd H.; Ciznar, Peter; Shcherbina, Anna; Kılıç, Sara Şebnem; Baumann, Ulrich; Seidel, Markus G.; Gennery, Andrew R.; Syczewska, Magorzata; Mikoluc, Bozena; Kalwak, Krzysztof; Styczynski, Jan; Pieczonka, Anna; Drabko, Katarzyna; Wakulinska, Anna; Gathmann, Benjamin; Albert, Michael H.; Skarzynska, Urszula; Bernatowska, Ewa; Inborn Errors Working Party Soc; KILIÇ GÜLTEKİN, SARA ŞEBNEM; Uludağ Üniversitesi/Tıp Fakültesi/Pediatrik İmmünoloji Anabilim Dalı.; AAH-1658-2021Purpose Nijmegen Breakage Syndrome (NBS) is a rare inherited condition, characterized by microcephaly, chromosomal instability, immunodeficiency, and predisposition to malignancy. This retrospective study, characterizing the clinical and immunological status of patients with NBS at time of diagnosis, was designed to assess whether any parameters were useful in disease prognosis, and could help determine patients qualified for hematopoietic stem cell transplantation.Methods The clinical and immunological characteristics of 149 NBS patients registered in the online database of the European Society for Immune Deficiencies were analyzed.Results Of the 149 NBS patients, 91 (61 %), of median age 14.3 years, remained alive at the time of analysis. These patients were clinically heterogeneous, with variable immune defects, ranging from negligible to severe dysfunction. Humoral deficiencies predisposed NBS patients to recurrent/chronic respiratory tract infections and worsened long-term clinical prognosis. Eighty malignancies, most of lymphoid origin (especially non-Hodgkin's lymphomas), were diagnosed in 42 % of patients, with malignancy being the leading cause of death in this cohort. Survival probabilities at 5, 10, 20 and 30 years of age were 95, 85, 50 and 35 %, respectively, and were significantly lower in patients with than without malignancies.Conclusions The extremely high incidence of malignancies, mostly non-Hodgkin's lymphomas, was the main risk factor affecting survival probability in NBS patients. Because treatment of NBS is very difficult and frequently unsuccessful, the search for an alternative medical intervention such as hematopoietic stem cell transplantation is of great clinical importance.Item Prognostic discrimination based on the EUTOS long-term survival score within the International Registry for Chronic Myeloid Leukemia in children and adolescents(Ferrata Storti Foundation, 2017-08-17) Millot, Frédéric; Guilhot, Joëlle; Suttorp, Meinolf; Sedlacek, Petr; De Bont, Eveline; Li, Chi Kong; Kalwak, Krzysztof; Lausen, Birgitte; Culic, Srdjana; Dworzak, Michael; Kaiserova, Emilia; De Moerloose, Barbara; Roula, Farah; Biondi, Andrea; Baruchel, André; Güneş, Adalet Meral; Uludağ Üniversitesi/Tıp Fakültesi/Çocuk Hematoloji Anabilim Dalı.; 24072843300The EUTOS Long-Term Survival score was tested in 350 children with chronic myeloid leukemia in first chronic phase treated with imatinib and registered in the International Registry for Childhood Chronic Myeloid Leukemia. With a median follow up of 3 years (range, 1 month to 6 years) progression and/or death (whichever came first) occurred in 23 patients. For the entire cohort of patients the 5-year progression-free survival rate was 92% (95% CI: 87%-94%) and the 5-year survival accounting for chronic myeloid leukemia deaths was 97% (95% CI: 94%-99%). Of the 309 patients allocated to low (n=199), intermediate (n=68) and high (n=42) risk groups by the EUTOS Long-Term Survival score, events (progression and/or death) occurred in 6.0%, 8.8% and 26.2%, respectively. Estimates of the 5-year progression-free survival rates according to these three risk groups were 96% (95% CI: 92%-98%), 88% (95% CI: 76%-95%) and 67% (95% CI: 48%-81%), respectively. Differences in progression-free survival according to these risk groups were highly significant (P < 0.0001, overall). The EUTOS Long-Term Survival score showed better differentiation of progression-free survival than the Sokal (<45 years), Euro and EUTOS scores in children and adolescents with chronic myeloid leukemia and should be considered in therapeutic algorithms.Item Prognostic discrimination of children and adolescents with chronic myeloid leukemia based on the EUTOS long term survival (ELTS) score(Amer Soc Hematology, 2016-12-02) Millot, Frederic; Guilhot, Joelle; Suttorp, Meinolf; Sedlacek, Petr; De Bont, Evelina S.; Li, Chi Kong; Kalwak, Krzysztof; Lausen, Birgitte; Culic, Srdjana; De Moerloose, Barbara; Biondi, Andrea; Baruchel, Andre; Güneş, Adalet Meral; Uludağ Üniversitesi/Tıp Fakültesi/Çocuk Hematoloji Anabilim Dalı.; EXD-8400-2022Publication Switch to subsequent line of treatment in children and adolescents with chronic myeloid leukemia (CML) treated with imatinib: Experience of the international registry for chronic myeloid leukemia in children and adolescents (I-CML-Ped Study)(Amer Soc Hematology, 2015-12-03) Millot, Frederic; Guilhot, Joelle; Suttorp, Meinolf; Meunier, Anne Sophie; Meral, Güneş Adalet; Sedlacek, Petr; de Bont, Eveline; Li, Chi Kong; Kalwak, Krzysztof; Lausen, Birgitte; Culic, Srdjana; De Moerloose, Barbara; Biondi, Andrea; Baruchel, Andre; MERAL GÜNEŞ, ADALET; Uludağ Üniversitesi/Tıp Fakültesi/Çocuk Hematoloji Anabilim Dalı.; IMO-4290-2023