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TBC regimens are considered as "reduced toxicity" and are increasingly employed in pediatric HSCT. In our center, we commonly use the combination of treosulfan-thiotepa-fludarabine and ATG for pediatric non-malignant diseases. As we often observe acute skin toxicities following this conditioning regimen, we conducted a prospective observational study to describe and characterize these toxicities. Fifteen pediatric patients undergoing HSCT for non-malignant diseases who were treated at Hadassah-Hebrew University Medical Center during 2015 were enrolled. A thorough dermatological assessment was done on days 0, 1, 7, and 14 from treatment initiation and included description of cutaneous reactions, measurement of BSA of affected skin, and response to local treatment. All the fifteen enrolled patients developed some degree of acute skin reaction. Cutaneous manifestations were variable and included erythematous patches in inguinal area and genitalia (80%), in neck and axillae (40%), diffuse hyperpigmentation (73%), erosions in inguinal area and buttock (47%), and xerosis and desquamation (40%). Average affected BSA reached 71.8%. Erosions were more prevalent in children younger than 2 years of age. The eruptions resolved without sequela in all patients and did not necessitate treatment other than topical agents. Observed extracutaneous toxicities included oral mucositis (40%), diarrhea (47%), and elevated liver enzymes (47%). TBC combined with thiotepa is highly toxic to the skin with various cutaneous manifestations. The toxicity resolves with no long-term sequela.

It is plausible that infections post-hematopoietic SCT play a role in the pathogenesis of BOS. A prospective study for children with history, questionnaire, examination, PFTs, and blood counts at one, three, six, nine, 12, 18, and 24 months post-SCT was conducted. Between September 2009 and September 2011 (n = 39), six developed BOS at 200 days (range 94-282), three patients had probable clinical respiratory infection, and all six had higher neutrophil count compared to non-BOS patients (4.7 vs. 2.4 at three months and 6.3 vs. 2.9 at six months ×10(9) /L, p = 0.03). Contribution of clinical and subclinical infection needs to be considered in the pathogenesis of BOS.

Gassas A, Zaidman I, Schechter T, Doyle J. Beyond gastrointestinal graft-versus-host disease. A lesson to learn. Pediatr Transplantation 2011: 15: E139–E141. © 2010 John Wiley & Sons A/S. Abstract: The authors present a child with severe GvHD of GI tract with typical presentation of ileus and abdominal pain. However, the severe GvHD has led to multiple intermittent intussusceptions with resultant ischemic segments of the small bowel leading to sub-acute mechanical intestinal obstruction. The clinical presentation of the mechanical intestinal obstruction was identical to the preceding ileus GvHD presentation. This has led to continuation of the conservative management with immunosuppressant therapy and delaying further investigation and surgical intervention. Once a barium study demonstrated the severe bowel constriction, laparoscopic surgical resection was necessary with excellent results. The resected small bowel showed multiple segments of ischemic ileitis leading to ulceration and severe stricture. The resection margins showed regenerating mucosa with no ulceration, but with focal evidence of mild GvHD.

The authors present a child with severe GvHD of GI tract with typical presentation of ileus and abdominal pain. However, the severe GvHD has led to multiple intermittent intussusceptions with resultant ischemic segments of the small bowel leading to sub-acute mechanical intestinal obstruction. The clinical presentation of the mechanical intestinal obstruction was identical to the preceding ileus GvHD presentation. This has led to continuation of the conservative management with immunosuppressant therapy and delaying further investigation and surgical intervention. Once a barium study demonstrated the severe bowel constriction, laparoscopic surgical resection was necessary with excellent results. The resected small bowel showed multiple segments of ischemic ileitis leading to ulceration and severe stricture. The resection margins showed regenerating mucosa with no ulceration, but with focal evidence of mild GvHD.