Patient 1:
A two-year-old boy, first and the only child of non-consanguineous
parents was diagnosed as SCID due to IL2RG mutation at 8 months with a
severe CMV pneumonia treated at ICU. Since a matched donor was not
available in the family, he underwent unconditioned haploidentical
transplantation from his father at 16 months. However, a primary graft
failure occurred and seven months later he retransplanted from an HLA
10/10 matched unrelated donor (MUD) following an RIC regimen consisting
of Treosulfan (42g/m2), Fludarabine
(150mg/m2) and ATG (20mg/kg). CSA was preferred for
GvHD prophylaxis. On post-transplant 7th day, grade I
skin aGvHD was detected that Methylprednisolone 2mg/kg/day was added to
the treatment. Since aGvHD of the skin did not respond to corticosteroid
but progressively exaggerated, infusions (3 x) of mesenchymal stem cells
and MMF were initiated followed by tacrolimus which was then switched to
sirolimus. On post-transplant 63rd day, during the
steroid tapering schedule, severe diarrhea (>2000cc/day)
and subsequently melena and fresh intestinal bleeding occurred. A Grade
IV GvHD was revealed by endoscopic and colonoscopic biopsies.
Generalized ulcers were found in the whole colon; however, the area of
bleeding could not be detected. Coagulation parameters were normal with
insignificant thrombocytopenia (platelet levels ranging from 50 to
120x109/L). However, erythrocyte transfusion twice a
day was required to maintain haemoglobin level. Oral budesonide and one
additional mesenchymal stem cells infusion were added to the treatment.
Furthermore, he did not respond to Tocilizumab (2x) (recombinant
humanized anti-interleukin-6 receptor (IL-6R) monoclonal antibody) which
was given in every two weeks.12-13 Severe and several
immunosuppressive agents refractory intestinal GvHD was controlled by 8
doses of alpha1-antitrypsin.(Table 1) After third dose of
alpha1-antitrypsin treatment, although diarrhea started to resolve,
intestinal bleeding persisted that Ankaferd Blood Stopper (a haemostatic
powder approved in Turkey) 14 initiated orally (2 ml
via nasogastric tube, twice a day for 7 days). After 4 days of
treatment, frequency and the amount of intestinal bleeding substantially
decreased while completely resolved following the first week. His oral
intake increased subsequently and he went home on post-transplant
139th day.