Multipl Miyelomda Otolog Kök Hücre Nakli Sonrası Gerçek Yaşam Sonuçları: Tek Merkezli Retrospektif Bir Çalışma

Abstract

Giriş: Yeni nesil tedavi ajanlarının yaygın şekilde kullanımına rağmen, otolog kök hücre nakli (ASCT), nakil için uygun multipl miyelom (MM) hastalarında tedavinin temel bileşenlerinden biri olmaya devam etmektedir. Randomize çalışmalar etkinliğini ortaya koymuş olsa da, gerçek yaşam verileri rutin klinik uygulamada sonuçları ve prognostik faktörleri değerlendirmek açısından önemlidir. Bu çalışmada, tek merkezde ASCT uygulanan MM hastalarının klinik özellikleri, nakil ilişkili sonuçları ve sağkalım verilerinin değerlendirilmesi amaçlanmıştır. Yöntemler: ASCT uygulanan 112 MM hastası retrospektif olarak analiz edildi. Başlangıç klinik ve laboratuvar özellikleri, indüksiyon tedavi rejimleri, nakil ile ilişkili değişkenler ve yanıt oranları değerlendirildi. Progresyonsuz sağkalım (PFS) ve genel sağkalım (OS) Kaplan–Meier yöntemi ile hesaplandı, prognostik faktörler ise Cox orantısal risk regresyon modeli ile analiz edildi. Bulgular: Ortanca yaş 60 olup hastaların %21,4’ü 65 yaş üzerindeydi. Bortezomib temelli indüksiyon tedavisi sonrasında hastaların çoğu nakil öncesinde çok iyi parsiyel yanıt veya daha iyi yanıt elde etti. Nakil sonrası 100. günde genel yanıt oranı %90’ın üzerinde olup hastaların %33’ünde tam yanıt saptandı. Medyan PFS 23,8 ay iken medyan OS’a ulaşılamadı. Uluslararası Evreleme Sistemi (ISS) evre II–III hastalık ve nakil öncesi parsiyel yanıt veya daha düşük yanıt düzeyi, hem PFS hem de OS için bağımsız olarak daha kötü prognoz ile ilişkili bulundu. Sonuç : Gerçek yaşam verilerimiz, yeni nesil ajanlar çağında da ASCT’nin önemli klinik fayda sağlamaya devam ettiğini göstermektedir. Nakil öncesi yanıt derinliği ve tanı anındaki hastalık evresi, uzun dönem sağkalım sonuçlarının en önemli belirleyicileri olarak öne çıkmaktadır.

Keywords

• Multiple myeloma
• Otolog kök hücre nakli
• Hastalıksız sağkalım
• Genel sağkalım

Supporting Institution

Bu çalışma için herhangi bir fon, hibe veya başka bir destek alınmamıştır.

Ethical Statement

Bu çalışma, Ondokuz Mayıs Üniversitesi Klinik Araştırmalar Etik Kurulu tarafından onaylanmıştır (Onay No: B.30.2.0DM.0.20.08/214; Karar No: 2023/135) ve Helsinki Bildirgesi ile sonraki revizyonlarına uygun olarak yürütülmüştür.

Clinical outcomes of autologous stem cell transplantation in multiple myeloma: A single-center experience

Abstract

Despite the widespread integration of novel therapeutic agents, autologous stem cell transplantation (ASCT) remains a cornerstone of treatment for transplant-eligible patients with multiple myeloma (MM). While randomized trials have established its efficacy, real-world data are essential for evaluating outcomes and prognostic factors in routine clinical practice. This study aimed to assess clinical characteristics, transplant-related outcomes, and survival in MM patients undergoing ASCT at a single center. We retrospectively analyzed 112 MM patients who underwent ASCT. Baseline clinical and laboratory features, induction regimens, transplant-related variables, and response rates were evaluated. Progression-free survival (PFS) and overall survival (OS) were estimated using the Kaplan–Meier method, and prognostic factors were analyzed using Cox proportional hazards regression models. The median age was 60 years, with 21.4% of patients aged over 65 years. Following bortezomib-based induction therapy, the majority of patients achieved very good partial response or better before ASCT. By day 100 post-transplant, the overall response rate exceeded 90%, with complete response achieved in 33% of patients. The median PFS was 23.8 months, while the median OS was not reached. International Staging System (ISS) stage II–III disease and a pre-transplant response of partial response or less were independently associated with inferior PFS and OS. Our real-world data confirm that ASCT continues to provide substantial clinical benefit in the era of novel agents. Depth of response prior to transplantation and disease stage at diagnosis remain the most important determinants of long-term survival outcomes.

Keywords

• Multiple myeloma
• Autologous stem cell transplantation
• Progression free survival
• Overall survival

Supporting Institution

No funding, grants, or other support was received for this study.

Ethical Statement

This study was approved by the Ondokuz Mayıs University Clinical Research Ethics Committee (Approval No: B.30.2.0DM.0.20.08/214; Decision No: 2023/135) and was conducted in accordance with the Declaration of Helsinki and its later amendments.

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