tacrolimus and Anemia--Sickle-Cell

Topics: Acute Disease; Anemia, Sickle Cell; Antisickling Agents; beta-Thalassemia; Bradycardia; Budd-Chiari Syndrome; Cardiopulmonary Resuscitation; Cholestasis, Intrahepatic; Glucocorticoids; Graft Rejection; Heart Arrest; Humans; Hydroxyurea; Immunosuppressive Agents; Liver Failure, Acute; Liver Transplantation; Male; Postoperative Complications; Prednisone; Tacrolimus; Young Adult

Unrelated donor (URD) hematopoietic cell transplantation (HCT) in children with sickle cell disease (SCD) is associated with a high incidence of rejection and graft-versus-host disease (GVHD). We report on the first 4 patients with severe SCD who underwent URD HCT using a novel myeloablative and immunosuppressive regimen composed of busulfan, fludarabine, and antithymocyte globulin with a single dose of post-transplant cyclophosphamide along with tacrolimus and mycophenolate mofetil for GVHD prophylaxis. Three patients engrafted and remain disease-free after a median follow-up period of 2.5 years. One patient had primary graft failure attributed to low stem cell content of the graft. Of interest, none of the engrafted patients developed acute or chronic GVHD. This preparative regimen along with the use of post-transplant cyclophosphamide offers a promising approach for unrelated donor transplants in patients with SCD and needs further corroboration in larger number of patients.

Topics: Adolescent; Anemia, Sickle Cell; Antilymphocyte Serum; Antineoplastic Combined Chemotherapy Protocols; Busulfan; Child; Child, Preschool; Cyclophosphamide; Female; Graft vs Host Disease; Hematopoietic Stem Cell Transplantation; Humans; Male; Mycophenolic Acid; Premedication; Tacrolimus; Tissue Donors; Transplantation, Homologous; Treatment Outcome; Vidarabine; Young Adult

The art of diagnosis consists in the recognition of signs and symptoms and their interpretation under the light of individual circumstances obtained from the patient's history. In medicine we encounter cases that are straightforward and cases that are complex as they are the result of a rare disease or of uncommon presentation of a common disorder. We present in this paper the case of a patient with a non-healing traumatic wound to her shin, which after unsuccessful treatments was biopsied and an unexpected diagnosis of necrobiosis lipoidica diabeticorum was made. This led to the institution of disease-targeted wound care leading to complete resolution in a short period of time.

Topics: Anemia, Sickle Cell; Female; Guidelines as Topic; Humans; Immunosuppressive Agents; Leg Injuries; Middle Aged; Necrobiosis Lipoidica; Skin Ulcer; Tacrolimus; Treatment Outcome; Wound Healing