Eculizumab and thrombotic microangiopathy after hematopoietic stem cell transplantation: A report on its efficacy and safety in two pediatric patients.

BACKGROUND: Thrombotic microangiopathy (TMA) is a severe complication after hematopoietic stem cell transplantation (HSCT), with the reported mortality rate in such cases usually reaching 90%. CASES: We report on two pediatric cases of patients successfully treated by eculizumab for severe HSCT-TMA, occurring in two girls (8.4 and 3.6 years). The first patient developed TMA with hematologic abnormalities and renal/pulmonary lesions after allogeneic HSCT for Philadelphia-positive acute lymphoblastic leukemia; she received eculizumab 7 months after HSCT, with a dramatic improvement of renal function. The second patient developed severe TMA (cardiac tamponade, renal failure requiring dialysis, gastritis) after autologous HSCT for metastatic neuroblastoma. She received eculizumab for 7 months, with a dramatic improvement of renal function. No side effects were observed. CONCLUSION: The use of eculizumab as first-line therapy in pediatric patients with severe HSCT-TMA with multisystemic lesions appears promising. Larger international studies are required to confirm its benefit and safety for this specific indication.

[Food protein-induced enterocolitis syndrome: A case report of diarrhea with hypovolemic shock and methemoglobinemia]. / Syndrome d'entérocolite induite par les protéines alimentaires (SEIPA) : à propos d'une observation de diarrhée avec choc hypovolémique et méthémoglobinémie.

We report on the case of a young infant with chronic diarrhea that worsened and turned into hypovolemic shock with methemoglobinemia. We underline and discuss the main features of food protein-induced enterocolitis syndrome (FPIES). FPIES is a non-IgE-mediated food allergy involving tumor necrosis factor-alpha (TNF-α). Many triggering foods exist but cow's milk, as in the case reported herein, is one of the most frequent. It can start early or be delayed and start around the average age of 5 months. Symptoms are nonspecific with diarrhea and vomiting, but in the presence of methemoglobinemia, the diagnosis must be seriously considered. The oral food challenge remains the gold standard to confirm the diagnosis if there is still a doubt. Treatment of FPIES associates emergency treatment of acute dehydration with the prevention of relapses by avoiding the suspected protein.