Outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome.

HLA-identical sibling donor transplantation remains the treatment of choice for Wiskott-Aldrich Syndrome (WAS). Since 1990, utilization of alternative donor sources has increased significantly. We report the hematopoietic cell transplantation (HCT) outcomes of 47 patients with WAS treated at a single center since 1990. Improved outcomes were observed after 2000 despite the increased number of alternative donors. Five-year OS improved from 62.5% (95% CI: 34.9% to 81.1%) to 90.8% (95% CI: 67.7% to 97.6%) for patients transplanted during 1990-2000 and 2001-2009, respectively. In multivariate analysis, transplant era significantly impacted OS (P=0.04), whereas age was only marginally significant (P=0.06, Cox proportional hazard analysis). No TRM occurred within the first 100 days among patients transplanted during 2001-2009 compared with 3/16 during 1990-2000, (P=0.03, Fisher's exact test). The extent of HLA mismatch did not significantly affect the incidence of acute GVHD, chronic GVHD or survival. Post-HCT autoimmune cytopenias were frequently diagnosed after 2001: 17/31 (55%) patients. Their occurrence was not associated with transplant donor type (P=0.53), acute GVHD (P=0.74), chronic GVHD (P=0.12), or post-transplant mixed chimerism (P=0.50).

Hematopoietic cell transplantation for hemophagocytic lymphohistiocytosis: a journey of a thousand miles begins with a single (big) step.

Hemophagocytic lymphohistiocytosis (HLH) is a rare, highly fatal disorder of uncontrolled inflammation, usually affecting infants. Significant progress in the treatment of this disorder has been achieved during the last decade, and outcomes for larger series of patients have been reported in recent years. Although medical therapy has advanced, hematopoietic cell transplantation remains the only curative therapy for patients with the familial form of this disorder. Unfortunately, these patients have demonstrated relatively poor post-transplant outcomes for a nonmalignant disorder, with approximately 30% mortality in the first 100 days. Early deaths were attributable to infection, GVHD, and unusually high rates of primary nonengraftment, venoocclusive disease and pneumonitis. In addition, a significant number of deaths were due to HLH reactivation, a unique complication seen in this patient group. In contrast, late complications were relatively infrequent and essentially all patients with durable engraftment remained in remission indefinitely. In this review, we will discuss recent progress in the transplant management of patients with HLH and potential future strategies, including the use of reduced intensity conditioning regimens.

Effects of etomidate and hypothermia on cerebral metabolism and blood flow in a canine model of hypoperfusion.

Etomidate is a nonbarbiturate hypnotic agent which, like the barbiturates, decreases the cerebral metabolic rate of oxygen consumption (CMRO2) 35-50%. The present studies assessed whether etomidate decreased CMRO2 through temperature-dependent mechanisms and whether the combination of etomidate and moderate hypothermia (28 degrees C) decreased CMRO2 more than hypothermia alone. Nineteen anesthetized dogs were treated with saline, etomidate (burst-suppressive doses), etomidate with hypothermia, or hypothermia alone. Etomidate did not affect (p > 0.05) the mean arterial pressure (MAP, mm Hg) but modestly lowered the heart rate [HR; 124 +/- 6 to 105 +/- 14, (mean +/- SEM); p < 0.05] whereas hypothermia (without or with etomidate) lowered (p < 0.05) both MAP (141 +/- 4 to 116 +/- 5 and 135 +/- 6 to 81 +/- 7) and HR (135 +/- 14 to 84 +/- 3 and 135 +/- 10 to 69 +/- 5, respectively). Etomidate administration did not result in a change (p > 0.05) in the esophageal, brain parenchymal, or subdural temperature. CMRO2 (ml/100 g/min) decreased (p < 0.05) during etomidate administration (3.2 +/- 0.4 to 1.7 +/- 0.2) and hypothermia (3.5 +/- 0.2 to 1.1 +/- 0.2), but the addition of etomidate to hypothermia did not further reduce CMRO2 in the animals (3.1 +/- 0.5 to 1.3 +/- 0.2) despite decreasing their brain hemispheric electrical activity from 9 +/- 1 Hz to a burst-suppressive state.(ABSTRACT TRUNCATED AT 250 WORDS)

Laryngeal compression and stridor as the presenting feature of ruptured thoracic aortic aneurysm. Report of two cases.

Two patients are reported in whom rupture of a thoracic aortic aneurysm presented with neck swelling and progressive stridor. Stridor occurred due to compression of the larynx by haematoma tracking up from the mediastinum. The presentation of thoracic aortic aneurysms and of rupture of thoracic aortic aneurysms are discussed with particular reference to mechanisms of cardiovascular and respiratory manifestations. The importance of early control of the airway is emphasised if there is to be any hope of survival from acute rupture.

Fibrous histiocytoma of the larynx.

Fibrous histiocytoma is uncommon in the head and neck region and rare in the larynx. Its behaviour is unpredictable. A case is reported in a 54-year-old male which was treated conservatively initially and which recurred several times over a seven year period. During that time the appearances of the lesion changed from those of a densely collagenous, cytologically benign tumour to those of a richly cellular and potentially malignant neoplasm. Laryngectomy was eventually undertaken.