Amaç: Ýmmün trombositopeni (ÝTP) ve çölyak hastalýðý (ÇH) arasýndaki iliþki hala belirsizliðini korumaktadýr. Bu çalýþmanýn amacý Ýtalyan
çocuklarýnda bu iki hastalýk arasýndaki iliþkiyi ortaya koymaktýr.
Gereç ve Yöntem: Bu çalýþma, 1 Ocak 2000’den 31 Aralýk 2019’a kadar ÝTP tanýsýna ek olarak ÇH tanýsý alan 28 çocuðun araþtýrýldýðý geriye dönük çok merkezli bir çalýþmadýr.
Bulgular: Hastalarýn %57,1’inde ilk taný ÝTP ve %32,1’inde ÇH idi. Üç hastada (%10,7) iki taný ayný anda konulmuþtu. Kohortumuzdaki tüm potansiyel ve sessiz ÇH olgularý “önce ÝTP” ve “eþzamanlý taný” gruplarýnda teþhis edildi. Tüm çocuklarda ÝTP hafifti ve ÇH tanýsý sýrasýnda trombositopenisi devam eden 8 çocuktan 2’sinde, glutensiz diyete baþladýktan sonra sýrasýyla 3 ve 5 ayda trombosit sayýlarýnda normalleþme (>100.000/μL) görüldü.
Sonuç: Bu çalýþma ÝTP’li çocuklarda gastrointestinal semptomlarýn varlýðýna bakýlmaksýzýn ÇH taramasýnýn yapýlmasý gerektiðini düþündürmektedir. Bazý hastalarda trombosit sayýlarýnýn glutensiz bir diyete baþladýktan sonra düzelebileceði akýlda bulundurulmalýdýr.

Objective: The association between celiac disease (CD) and immune thrombocytopenia (ITP) is still uncertain. The aim of this study was to characterize the coexistence of these two diseases in Italian children.
Materials and Methods: This is a retrospective multicenter study investigating the occurrence of CD in 28 children with ITP diagnosed from January 1, 2000, to December 31, 2019.
Results: The first diagnosis was ITP in 57.1% and CD in 32.1% of patients. In 3 patients (10.7%), the two diagnoses were simultaneous. All the potential and silent cases of CD in our cohort were diagnosed in the groups of “ITP first” and “simultaneous diagnosis”. In all children ITP was mild, and in 2 out of 8 not recovered from ITP at the time of CD diagnosis a normalization of platelet counts (>100,000/μL) occurred 3 and 5 months after starting a gluten-free diet, respectively.
Conclusion: We think that screening for CD should be considered in children with ITP regardless of the presence of gastrointestinal symptoms. Furthermore, some patients may recover from ITP after starting a gluten-free diet.

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Amaç: Primer immün trombositopeni (ÝTP), izole trombositopeni ile karakterize, edinsel bir otoimmün hastalýktýr. Birinci basamak tedaviler otoantikorlarý ve trombosit yýkýmýný inhibe etmeye odaklanýrken, ikinci ve üçüncü basamak tedaviler arasýnda splenektomi ve trombopoietin reseptör agonistleri bulunur. Bu çalýþmada, ÝTP’de ikinci basamak tedaviler olarak splenektomi ve eltrombopagýn etkinlik vetoksisitelerinin karþýlaþtýrýlmasý amaçlanmýþtýr.
Gereç ve Yöntem: 2015-2020 yýllarý arasýnda ÝTP tanýsý alan ve takip edilen hastalarý retrospektif olarak analiz ettik. Splenektomi yapýlan ve 2. veya daha ileri basamaklarda eltrombopag tedavisi alan hastalar çalýþmaya dahil edildi. Alt grup analizleri için, hastalar ikinci veya üçüncü tedavi hattýnda eltrombopag alýp almadýklarýna göre gruplandýrýldý.
Bulgular: Splenektomi grubunda 38 hasta, eltrombopag grubunda 47 hasta vardý. Splenektomi ve eltrombopag gruplarýndaki hastalarýn ortalama yaþý sýrasýyla 43,2 ve 50,5’di. Splenektomi kolunda yanýt süresi anlamlý olarak daha kýsaydý (p=0,001). Bununla birlikte, 3., 6., 12. ve 24. aylardaki yanýt oranlarý, toplam yanýt süresi ve yan etkiler açýsýndan gruplar arasýnda istatistiksel olarak anlamlý fark bulunmadý. Birinci, 3., 6., 12. ve 24. aylardaki yanýt oranlarý ve toplam yanýt süresi, eltrombopag alt gruplarý arasýnda istatistiksel olarak anlamlý deðildi. Eltrombopag tedavisi medyan 54,1 ay (1-151) sonra 20 hastada kesildi. Bunlardan 12 hastada (%60) yanýt kaybý yaþanmadý.
Sonuç: Splenektomi ve eltrombopag kollarý karþýlaþtýrýldýðýnda yanýt alma süresi splenektomi lehine olsa da, genel yanýt oranlarý ve 2. yýldaki
yanýt oranlarý dikkate alýndýðýnda bu avantaj ortadan kalkmaktadýr. Ýkinci veya 3. basamak tedavide eltrombopag kullanýlmasý, yanýt alma süresi açýsýndan herhangi bir fark oluþturmamaktadýr.

Objective: Primary immune thrombocytopenia (ITP) is an acquired autoimmune disease characterized by isolated thrombocytopenia. While first-line treatments focus on inhibiting autoantibodies and platelet destruction, second- and third-line treatments include splenectomy and thrombopoietin receptor agonists. In this study, we aimed to compare the efficiency and toxicities of splenectomy and eltrombopag as second-line treatments in ITP.
Materials and Methods: We retrospectively analyzed patients who were diagnosed with ITP and followed between 2015 and 2020. Patients who underwent splenectomy or received eltrombopag treatment as second-line or further therapy were included. For subgroup analyses, patients were further stratified according to whether they received eltrombopag in the second or third line of treatment.
Results: There were 38 patients in the splenectomy group and 47 patients in the eltrombopag group. The mean age of patients in the splenectomy and eltrombopag groups was 43.2 and 50.5 years, respectively. Time to response was significantly shorter in the splenectomy arm (p=0.001). However, response rates at the 3rd, 6th, 12th, and 24th months did not exhibit a statistically significant difference between groups; nor did total duration of response and adverse events. Response rates at the 1st, 3rd, 6th, 12th, and 24th months and the total duration of response did not exhibit a statistically significant difference between eltrombopag subgroups. Eltrombopag treatment was ceased for 20 patients after a median of 54.1 months (range: 1-151). Among them, 12 patients (60%) did not experience a loss of response.
Conclusion: Comparing the splenectomy and eltrombopag arms, even though time to achieve response was in favor of the splenectomy group, this advantage disappeared when overall response rates and response rate at the 2nd year were considered. Using eltrombopag in the second or third line of therapy does not yield any difference in terms of time to achieving response.

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Amaç: Bu çalýþma, 2016 Dünya Saðlýk Örgütü sýnýflamasýna göre myelodisplazi iliþkili deðiþiklikler gösteren akut myeloid löseminin (AML-MRC) klinik özelliklerini ve 60-75 yaþ arasý AML-MRC hastalarýnda tercih edilen tedaviyi araþtýrmayý amaçlamýþtýr.
Gereç ve Yöntem: AML-MRC’li 190 hasta ve baþka þekilde tanýmlanmamýþ AML’li (AML-NOS) 667 hasta arasýndaki klinik farklýlýklarý geriye dönük olarak analiz ettik. Ayrýca 60-75 yaþ arasý AML-MRC’li hastalar arasýnda farklý terapötik rejimleri karþýlaþtýrdýk.
Bulgular: AML-NOS ile karþýlaþtýrýldýðýnda, AML-MRC’li hastalar, daha kötü genel saðkalým (GS) (9,22’ye karþý 13,6 ay; p<0,001) ve daha düþük tam remisyon oranlarý ile (%65,3’e karþý %76,2; p=0,005) önemli ölçüde farklý klinik özelliklere sahipti. Tüm grup (hem AML-MRC hem de AML-NOS’li hastalar) için yapýlan çok deðiþkenli analiz, AML-MRC’nin baðýmsýz prognostik faktör olduðunu gösterdi (p=0,002). AML-MRC’li 190 hasta için yapýlan ek çok deðiþkenli analiz, yaþýn (p<0,001) ve laktat dehidrogenaz düzeyinin (p=0,031) baðýmsýz prognostik faktör olduðunu gösterdi. IA/DA rejimi [idarubisin ve sitarabin (IA) veya daunorubisin ve sitarabin (DA)] ile karþýlaþtýrýldýðýnda, DAC+CAG rejimi [desitabin ve yarým doz CAG rejimi (sitarabin, aklarubisin ve granülosit koloni uyarýcý faktör)] 60-75 yaþ arasý ve olumsuz risk grubuna dahil olan hastalarda daha iyi GS ile iliþkili (4,5’e karþý 6,2 ay; p=0,021) bulundu.
Sonuç: AML-MRC olgularý, AML-NOS’ye kýyasla daha kötü klinik sonuçlar sergilemiþtir. IA/DA rejimi ile karþýlaþtýrýldýðýnda, DAC+CAG rejimi, olumsuz risk grubundaki 60-75 yaþ AML-MRC’li hastalar için optimal seçim olarak bulunmuþtur.

Objective: This study aimed to investigate the clinical characteristics of acute myeloid leukemia with myelodysplasia-related changes (AMLMRC)
according to the 2016 World Health Organization classification and the preferred therapy for patients with AML-MRC aged 60-75 years.
Materials and Methods: We retrospectively analyzed differences in clinical data among 190 patients with AML-MRC and 667 patients with AML not otherwise specified (AML-NOS). We also compared different therapeutic regimens among patients with AML-MRC aged 60-75 years.
Results: Compared with AML-NOS, patients with AML-MRC had significantly different clinical characteristics as well as worse overall survival (OS) (9.2 vs. 13.6 months; p<0.001) and complete remission rates (65.3% vs. 76.2%; p=0.005). Multivariate analysis performed for the whole group (patients with both AML-MRC and AML-NOS) showed that AML-MRC was the independent prognostic factor (p=0.002). Additional multivariate analysis performed for 190 patients with AML-MRC indicated that age (p<0.001) and lactate dehydrogenase (p=0.031) were independent prognostic factors. Compared with the IA/DA regimen [idarubicin and cytarabine (IA) or daunorubicin and cytarabine (DA)], the DAC+CAG regimen [decitabine and half-dose CAG regimen (cytarabine, aclarubicin, and granulocyte colony-stimulating factor)] was associated with better OS (4.5 vs. 6.2 months; p=0.021) in patients aged 60-75 years and categorized into the unfavorable risk group.
Conclusion: AML-MRC cases exhibited worse clinical outcomes compared to AML-NOS. Compared to the IA/DA regimen, the DAC+CAG regimen was the optimal choice for patients with AMLMRC in the unfavorable risk group and aged 60-75 years.

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Amaç: Paroksismal noktürnal hemoglobunüri (PNH) hastalarýnýn tedavisinde kompleman sisteminin deðiþik basamaklardaki inhibisyonu umut vadedici bir tedavi yöntemidir. Ancak hastalýðýn belirti ve bulgularýný kontrol etmekte etkin bir tedavi yöntemi olsa da küratif bir tedavi seçeneði deðildir. Özellikle kompleman inhibisyonuna raðmen devam eden hemolizi, replasman ihtiyacý ve kemik iliði yetmezliði ile birlikteliði olan hasta grubunda halen tek küratif tedavi seçeneði allojeneik kök hücre naklidir (KHN). Bu çalýþmamýzda aplastik anemi ile birlikteliði olan (PNH-AA) ve olmayan PNH hastalarýnda yapýlmýþ allojeneik KHN’nin sonuçlarýný deðerlendirmeyi amaçladýk.
Gereç ve Yöntem: On ayrý merkezde PNH/PNH-AA tanýsý olup allojeneik KHN yapýlan toplam 35 hasta retrospektif olarak tarandý.
Bulgular: On altý (%45,7) PNH ve 19 (%54,3) PNH-AA hastasý çalýþmaya dahil edildi. Hastalarýn ortanca yaþý 32 (18-51) idi. Hastalardaki saðkalým analizlerine baktýðýmýzda; 2 yýllýk genel saðkalým (GS) ve graft versus host hastalýksýz ve graft yetmezliksiz saðkalým (GFFS) oranlarý sýrasýyla %81,2 ve %78,1 idi. Ýki yýllýk saðkalým PNH ve PNH-AA hastalarýnda sýrasýyla %81,3 ve %79,9 olarak hesaplandý ve gruplar arasýnda istatistiksel bir fark tespit edilmedi (p=0,87). Tam uyumlu akraba donör ve tam uyumlu akraba dýþý donör alt gruplarý karþýlaþtýrýldýðýnda 2 yýllýk OS ve GFFS oranlarýnda istatistiksel fark gözlenmedi.
Sonuç: Seçilmiþ PNH ve PNH-AA hasta grubunda akraba veya akraba dýþý tam uyumlu donör ile yapýlan allojeneik KHN uygun bir seçenektir.
Özellikle refrakter hemolizi ve replasman ihtiyacý devam eden ve kemik iliði yetmezliði bulgularý olan hasta grubunda allojeneik KHN bir tedavi seçeneði olarak deðerlendirilmelidir.

Objective: Although inhibition of the complement system at different steps is a promising therapy modality in patients with paroxysmal nocturnal hemoglobinuria (PNH), allogeneic hematopoietic stem cell transplantation (HCT) is still the only curative therapy, especially for patients with intractable hemolysis or bone marrow failure. The aim of this study is to evaluate the outcomes of allogeneic HCT in PNH patients with aplastic anemia (PNH-AA) or without.
Materials and Methods: Thirty-five PNH/PNH-AA patients who were treated with allogeneic HCT in 10 transplantation centers in Turkey were retrospectively analyzed.
Results: Sixteen (45.7%) and 19 (54.3%) patients were diagnosed with classical PNH and PNH-AA, respectively. The median age of the patients was 32 (18-51) years. The 2-year overall survival (OS) rate and rate of graft-versus-host disease-free, failure-free survival (GFFS) was 81.2% and 78.1%, respectively. The 2-year OS in cases of classical PNH and PNH-AA was 81.3% and 79.9%, respectively (p=0.87), and 2-year GFFS in cases of PNH and PNH-AA was 79% and 76% (p=0.977), without statistical significance. The OS and GFFS rates also did not differ between transplantations with matched sibling donors (MSDs) and matched unrelated donors (MUDs).
Conclusion: Allogeneic HCT with MSDs or MUDs is a good option for selected patients with classical PNH and PNH-AA. In particular, patients with debilitating and refractory hemolysis and patients with bone marrow failure might form an excellent group of candidates for allogeneic HCT.

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Amaç: Otolog kök hücre transplantasyonu (OKHT), relaps/refrakter lenfoma hastalarý için önemli ve potansiyel olarak küratif bir tedavi yöntemidir. Yetersiz mobilizasyon ve periferik kök hücrelerin toplanmasý OKHT’nin gerçekleþtirilmesi için büyük bir engel olabilir. Bu çalýþmanýn amacý lenfoma hastalarýnda mobilizasyon baþarýsýzlýðýný etkileyebilecek faktörleri deðerlendirmektir.
Gereç ve Yöntem: Hacettepe Üniversitesi Týp Fakültesi Kemik Ýliði Nakil Merkezi’nde 2000-2018 yýllarý arasýnda Hodgkin ve non-Hodgkin lenfoma tanýsý alan ve sonrasýnda kök hücre mobilizasyonu yapýlan 87 hasta deðerlendirildi.
Bulgular: Bu çalýþmaya toplam 87 hasta dahil edildi. Seksen yedi hastanýn 66’sýnda (%75,9) ilk mobilizasyon denemesi baþarýlý oldu. 66 hastada (9,5±8,1) ilk aferezde yeterli (≥2x106/kg) CD34+ hücre toplandý. Seksen yedi kiþiden 21’i (%24,1) için ilk mobilizasyon denemesi baþarýsýz oldu. Bu nedenle bu hastalara pleriksafor ile (5,5±3,3) ikinci bir mobilizasyon denemesi yapýldý. Ýlk mobilizasyonda baþarýlý olan hastalarda CD34+ hücre sayýsý anlamlý olarak yüksekti (p=0,002).
Sonuç: Mobilizasyon öncesi trombosit sayýsý yüksek olan hastalarda ve kemoterapi bazlý mobilizasyon protokolleri alan hastalarda ilk mobilizasyon denemesinin baþarý oraný daha yüksek bulundu. Ýlk denemede mobilizasyon baþarýsýzlýðý olan hastalarda daha sonraki
mobilizasyonda pleriksafor kullanýlmýþ ve bu hastalarda OKHT için yeterli miktarda kök hücre mevcuttu. Mobilizasyon baþarýsýzlýðýný öngören parametreler, ilk mobilizasyon giriþimi sýrasýnda bu tür ajanlarýn önleyici, daha uygun maliyetli kullanýmýna izin verecektir; ancak, mobilizasyon baþarýsýzlýðý için risk faktörleri hala net deðildir.

Objective: Autologous stem cell transplantation (ASCT) is a significant and potentially curative treatment modality for patients with relapsed/refractory lymphoma. Insufficient mobilization and harvest of peripheral stem cells can be a major obstacle for performing ASCT. The aim of this study was to evaluate the factors which might influence mobilization failure in patients with lymphoma.
Materials and Methods: Eighty-seven patients with diagnosed non-Hodgkin and Hodgkin lymphoma who underwent stem cell mobilization after at Hacettepe University Medical School, Bone Marrow Transplantation Center, Turkey, between the years of 2000 and 2018 were evaluated.
Results: A total of 87 patients were included in this study. In 66 of 87 patients (75.9%) first mobilization trial was successful. Adequate (≥2 × 106/kg) CD34+ cells were collected at first apheresis in 66 patients (9.5±8.1). In 21 of 87 (24.1%) first mobilization trial was unsuccessful. Therefore, a second mobilization trial was made to these patients with plerixafor (5.5±3.3). The number of CD34+ cell was significantly higher in patients who were successful in the first mobilization (p=0.002).
Conclusion: The success rate of the first mobilization trial was found to be higher in patients with high platelet counts before mobilization and patients who received chemotherapy-based mobilization protocols. In the patients who had mobilization failure in the first trial, plerixafor was used in a later mobilization, and those patients had an adequate amount of stem cells for ASCT. Parameters predicting mobilization failure would allow for preemptive, more cost-effective use of such agents during the first mobilization attempt; however, risk factors for mobilization failure are still not clear.

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Amaç: Sürekli artan saðlýk harcamalarý jenerik moleküllerin kullanýmýna yol açmaktadýr ve bortezomibin jenerik molekülleri uzun süredir kullanýlmaktadýr. Bu çalýþmanýn amacý, yeni taný konmuþ multipl myelom (MM) hastalarýnda jenerik bortezomibin etkinliðini, yan etkilerini ve güvenilirliðini retrospektif olarak incelemektir.
Gereç ve Yöntem: Tek merkezli çalýþmamýzda birinci veya ikinci basamak tedavide dört kür bortezomib alan 95 hastanýn verileri geriye dönük olarak incelendi. Tedavi yanýtlarý, yan etkiler ve progresyonsuz saðkalým (PFS) oranlarý hesaplandý ve karþýlaþtýrýldý.
Bulgular: Doksan beþ hastanýn 42’si orijinal molekülü, 53’ü eþdeðer molekülü kullanmýþtýr. Epidemiyolojik veriler, MM tipleri, genetik risk gruplarý, taný anýndaki laboratuvar deðerleri ve bortezomib tedavi sýralarý (birinci sýra veya ikinci sýra olarak) deðerlendirildiðinde iki grup arasýnda istatistiksel fark olmadýðý izlendi. Uluslararasý Myelom Çalýþma Grubu kriterlerine göre yanýt oranlarý deðerlendirildiðinde anlamlý bir fark bulunmadý (p=0,42). Kýsmi yanýt ve daha yüksek yanýt oranlarý benzerdi (%81’e karþý %79,2, p=0,84). PFS süreleri orijinal molekül için 42,8 ay ve eþdeðer molekül için 37,8 ay olarak hesaplandý (p=0,68). Yan etkiler tüm hastalarýn %44,2’sinde görüldü ve en sýk görülen yan etkiler nöropati, sitopeni ve enfeksiyondu. Bu yan etkilerin görülme oranlarý iki grupta benzerdi (p=0,55).
Sonuç: Bu retrospektif çalýþma, kapsamý sýnýrlý olmakla birlikte, orijinal bortezomib molekülünü eþdeðeri ile karþýlaþtýran ilk çalýþmadýr. Tedavi
yanýtlarý, PFS veya yan etkiler açýsýndan iki grup arasýnda istatistiksel fark izlenmemiþtir. Ancak geniþ çaplý deðerlendirmeler bu konuda
daha fazla veri elde edilmesine yardýmcý olacaktýr.

Objective: Constantly increasing health expenditures lead use of generic molecules and generics of bortezomib have been used for a long time. The aim of this study is to retrospectively examine the effectiveness, side effects and reliability of generic bortezomib in newly diagnosed multiple myeloma (MM) patients.
Materials and Methods: The data of 95 patients who received four cycles of bortezomib as a first- or second-line therapy in a single center were retrospectively recorded. Treatment responses, side effects and progression free survival (PFS) rates have been calculated and compared.
Results: Of the 95 patients, 42 used the original and 53 used the generic molecule. Epidemiological data, MM types, genetic risk groups, laboratory values at diagnosis and bortezomib treatment lines (as a first line or second) were evaluated, and there was no statistical difference between the two groups. When the response rates were evaluated with International Myeloma Working Group (IMWG) criteria, there were no significant difference (p: 0,42). Partial response (PR) and above response rates were similar (%81 vs %79,2 p: 0,836). PFS values were 42.8 months in the original and 37.8 months in the generic molecule group (p: 0,68). Side effects were seen in 44,2% of patients, and the most common side effects were neuropathy, cytopenias and infections. They were similar in both groups (p: 0,67).
Conclusion: Although this retrospective study is limited, it is the first study comparing the original molecule of bortezomib with its generic. There was no statistical difference between the two groups in terms of treatment responses, PFS times and side effects. However, large-scale evaluations can help obtain healthier data on this subject.

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Proteozom inhibitörleri (PÝ) multipl myelom tedavilerinin integral parçalarýndandýr. Çevresel nöropatiler (ÇN) PÝ’nin iyi bilinen bir sonucudur, bortezomib (BTZ) gibi önceki jenerasyonlarla daha sýk bildirilmiþtir. Yeni jenerasyon PI olan karfilzomib (KFZ) ve iksazomib (IZB) ile geliþen ÇN riskini vurgulayan çalýþmalar yetersizdir. Bu çalýþmada, Gýda ve Ýlaç Ýdaresi Olumsuz Olay (OO) Raporlama Sistemini (FAERS) kullanarak her üç PI ile karþýlaþýlan ÇN raporlarý deðerlendirildi. Sinyal orantýsýzlýk analizi, raporlama olasýlýk oraný (ROO) %95 güven aralýðý (GA) kullanýlarak yapýldý. ÇN; KFZ, IZB, BTZ ile sýrasýyla OO’nun toplamýnda %2,1, %5,0, ve %10,9’unda rapor edilmiþti. BTZ, KFZ, ve IZB’ye ikincil ÇN için ROO (%95 GA) sýrasýyla 34,10 (32,76-35,49), 6,37 (5,50-7,37), ve 14,97 (13,63-16,44) idi. KFZ ve IZB ile, BTZ ile karþýlaþtýrýldýðýnda daha az ÇN rapor edilmiþti, ROO sýrasýyla 0,19 (0,16-0,22) ve 0,48 (0,43-0,54) idi.

Proteasome inhibitors (PIs) are an integral component of multiple myeloma therapies. Peripheral neuropathy (PN) is a well-known consequence of PIs, most frequently reported with earlier generations such as Bortezomib (BTZ). There is a paucity of data highlighting the risk of developing PN in the newer generation PIs Carfilzomib (CFZ) and Ixazomib (IZB). This study queried reports of PN reported with all three PIs using the Food and Drug Administration (FDA) Adverse Event (AE) Reporting System (FAERS). Signal disproportionality analysis was reported using a reporting odds ratio (ROR) and 95% confidence interval (CI). PN was reported in a total of 2.1%, 5.0%, and 10.9% of AE with CFZ, IZB, and BTZ, respectively. The ROR (95% CI) for peripheral neuropathy secondary to BTZ, CFZ, and IZB was 34.10 (32.76-35.49), 6.37 (5.50- 7.37), and 14.97 (13.63-16.44) respectively. Compared to BTZ, CFZ and IZB have a lower rate of reported PN, with RORs of 0.19 (0.16-0.22) and 0.48 (0.43-0.54), respectively.

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