The Treatment of Steroid-Refractory Severe Gastrointestinal Acute Graft-Versus-Host Disease in Children after Allogeneic Hematopoietic Stem Cell Transplantation: A Single-Centre Experience

Öz

Acute graft-versus-host disease is a common complication of allogeneic hematopoietic stem cell transplantation and is a major cause of morbidity and mortality. Systemic steroid therapy is the first-line treatment for acute graft-versus-host disease, although about half of patients will become refractory to treatment. We aimed to evaluate treatment options by reviewing available alternatives for patients with steroid-refractory acute graft-versus-host disease by comparing data from recently published studies. We retrospectively studied the safety and efficacy of treatment in 22 children with steroid‐dependent/refractory acute GVHD between the years 2010 and 2023. Seven (31.8%) out of 22 patients with acute graft-versus-host disease were still alive. The seven surviving patients have been followed for an average of 1141 (±403) days. 15 non-responders with grade III/IV acute graft-versus-host disease died from causes associated with acute graft-versus-host disease and/or other conditions. Among 15 patients who died, the number of patients who received all three treatments, MSC, ECP and infliximab, was 5 (33%). The most common cause of death was infection (8 cases in 15 patients, 53.3%). Other causes of death were gastrointestinal hemorrhages (n=5, 33.3%), and intracranial hemorrhages (n=2, 13.3%). When factors causing morbidity were evaluated, it was observed that three patients developed posterior reversible encephalopathy syndrome, two patients developed hepatic veno-occlusive disease, and one patient developed hypertension. The estimated probability of survival after 1 year was 31.8%, and the median survival was 655 days. We believe that it would be crucial to show the safety and efficacy of novel treatments in comprehensive, randomized clinical trials.

Anahtar Kelimeler

• Allogeneic hematopoietic stem cell transplantation
• graft-versus-host disease
• pediatric.

Allojeneik Hematopoietik Kök Hücre Transplantasyonu Sonrası Çocuklarda Steroide Dirençli Şiddetli Gastrointestinal Akut Graft-Versus-Host Hastalığının Tedavisi: Tek Merkez Deneyimi

Öz

Akut graft-versus-host hastalığı, allojeneik hematopoietik kök hücre naklinin yaygın bir komplikasyonu olup önemli bir morbidite ve mortalite nedenidir. Sistemik steroid tedavisi, akut graft-versus-host hastalığı için ilk basamak tedavidir, ancak hastaların yaklaşık yarısı tedaviye direnç gösterebilmektedir. Akut graft-versus-host hastalığı için güvenli ve etkili tedavilerin keşfedilmesi, allojeneik kök hücre nakli yapılan hastaların sayısı arttıkça, özellikle durumu sistemik steroid tedavisine dirençli hale gelenler için daha önemli hale gelecektir. Yakın zamanda yayınlanmış çalışmalardan elde edilen verileri karşılaştırarak, steroide dirençli akut graft-versus-host hastalığı olan hastalar için mevcut alternatifleri gözden geçirerek tedavi seçeneklerini değerlendirmeyi amaçladık. 2010-2023 yılları arasında steroide bağımlı/refrakter akut graft-versus-host hastalığı olan 22 çocukta tedavinin güvenliğini ve etkinliğini retrospektif olarak inceledik. Akut graft-versus-host hastalığı olan 22 hastanın yedisi (%31,8) hayatta kaldı. Hayatta kalan hastaların takip gün sayısı ortalama 1141 (±403) idi. Evre III/IV akut graft-versus-host hastalığına sahip, tedaviye yanıt vermeyen 15 hasta farklı nedenlerden dolayı öldü. En sık ölüm nedeni enfeksiyondu (16 hastada 8 vaka, %53,3). Diğer ölüm nedenleri arasında mide-bağırsak kanaması (n=5, %33,3) ve kafa içi kanama (n=2, %13,3) yer aldı. Ayrıca morbiditeye neden olan faktörler değerlendirildiğinde üç hastada PRES sendromu, iki hastada hepatik veno-okluzif hastalık ve bir hastada hipertansiyon geliştiği görüldü. 1 yıl sonra tahmini hayatta kalma olasılığı %31,8 ve ortalama hayatta kalma süresi 655 gündü. Steroide dirençli akut graft-versus-host hastalığında kombinasyon tedavi yöntemlerinin etkisini retrospektif olarak değerlendirdik. Yeni tedavilerin güvenliğini ve etkinliğini kapsamlı, randomize klinik araştırmalarla göstermenin önemli olacağını düşünüyoruz

Anahtar Kelimeler

• Allojeneik Hematopoietik Kök Hücre Transplantasyonu
• Akut Graft-Versus-Host Hastalığı
• Pediatrik

Kaynakça

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