Langerhans Cell Histiocytosis, a Rare Disease in Adults: 22 Years of Single Center Experience

Öz

Langerhans cell histiocytosis (LCH) is a rare, multisystemic, and heterogeneous disease seen in adults. There is very little data on how to approach and the treatment of the adult patient. In this study, we retrospectively analyzed 16 adult patients with LCH who were referred to our center between March 2000 and March 2022. 68.7% of the patients were male, and the median age was 30.5 years. At the time of diagnosis, 50% of the patients had a single site, and 50% had multisystemic (MS). Risk organ involvement was present in 3 patients (18.75%). The most involved areas were bone and lung (43.75%), followed by the central nervous system (CNS) (31.25%). All eight patients with indications for systemic treatment were given vinorelbine and steroid treatment in the first line. Other patients underwent surgical excision or radiotherapy. The response rate to treatment was 37.5%, and the partial response rate was 37.5%. 3 (18.75%) of the patients relapsed. All patients with single-site involvement survived and did not relapse. Eleven patients are alive. Median overall survival (OS) was not achieved; 3-year OS was 79.8%. Survival of patients over 30 years of age was found to be lower. However, this difference was not statistically significant (p=0.2). Those with MS had worse median OS data (48 months vs. unavailable, p=0.02). Age at diagnosis over 30 and CNS involvement was associated with lower OS, but this was not statistically significant. Rapid disease progression or refractoriness to treatment was observed in patients with spleen and liver involvement. Especially in patients with poor prognostic features, vinblastine/steroid-based treatments may be insufficient in primary care. Multicenter, prospective studies are needed better to determine the clinical and prognostic features of adult LCH and to develop appropriate treatment strategies.

Anahtar Kelimeler

• Adult
• langerhans cell histiocytosis
• BRAF

Erişkinde Nadir Hastalıklardan Langerhans Hücreli Histiyositoz: 22 Yıllık Tek Merkez Deneyimi

Öz

Langerhans hücreli histiyositoz (LHH) erişkinde nadir rastlanılan multi-sistemik (MS) ve heterojen bir hastalıktır. Erişkin hastaya yaklaşım ve tedaviyle ilgili veri oldukça azdır. Bu çalışmada Mart 2000 ile Mart 2022 tarihleri arasında merkezimize sevk edilen LHH’li 16 erişkin hastayı retrospektif olarak inceledik. Hastaların %68,7’si erkek ve ortanca yaş 30,5 yıl idi. Tanıda hastaların %50’sinde tek bölgede, %50’sinde MS tutulum vardı. Riskli organ tutulumu 3 hastada (%18,75) mevcuttu. En çok tutulan alanlar kemik ve akciğer (%43.75), sonrasında ise santral sinir sistemi (SSS) (%31,25) idi. Sistemik tedavi endikasyonu olan 8 hastanın tamamına birinci sırada vinorelbin ve steroid tedavisi verildi. Diğer hastalara cerrahi eksizyon veya radyoterapi uygulandı. Tedaviye tam yanıt oranı %37.5, kısmi yanıt oranı %37.5 idi. Hastaların 3’ü (%18,75) nüks etti. Tek bölge tutulumu olan hastaların hepsi hayatta ve nüks gelişmedi. On bir hasta ise halen hayattadır. Median genel sağkalıma (OS) ulaşılamadı, 3 yıllık OS %79.8 idi. Otuz yaş üzerindeki hastaların sağkalımı daha düşük bulundu. Ancak bu fark istatistiksel olarak anlamlı değildi (p=0.2). MS hastalığı olanların median OS verileri daha kötüydü (48 aya karşı ulaşılamadı, p=0.02). Tanı yaşının 30’un üzerinde olması ve SSS tutulumu bulunması daha düşük OS ile ilişkiliydi ancak bu istatistiksel olarak anlamlı değildi. Dalak ve karaciğer tutulumu olan hastalarda hızlı hastalık progresyonu ya da tedaviye refrakterlik izlendi. Özellikle kötü prognostik özellikleri olan hastalara birinci basamakta vinblastin/steroid bazlı tedaviler yetersiz kalabilir. Erişkin LHH’da klinik ve prognostik özelliklerinin daha iyi belirlenebilmesi, uygun tedavi stratejilerinin geliştirilmesi için çok merkezli, prospektif çalışmalara ihtiyaç vardır.

Anahtar Kelimeler

• Erişkin
• langerhans hücreli histiyositoz
• BRAF.

Kaynakça

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