In this issue of Blood, Touzot et al report that autologous gene therapy/hematopoietic stem cell transplantation (HSCT) for infants with X-linked severe combined immune deficiency (SCID-X1) lacking a matched sibling donor may have better outcomes than haploidentical (haplo) HSCT. Because gene therapy represents an autologous transplant, it obviates immune suppression before and after transplant...

Mantle cell lymphoma (MCL) is an uncommon non-Hodgkin lymphoma with a heterogeneous natural history. Significant strides have been made in the management of MCL. Clinical follow-up exceeds a decade with long-term remission durations in some patients. Modern induction strategies employ rituximab; a cyclophosphamide, doxorubicin, vincristine, and prednisone (CHOP)-like backbone; and cytarabine (i...

Neurological complications are quite frequent in stem cell transplant (SCT) recipients. Major causes are conditioning regimen toxicity, metabolic and electrolyte disturbances, viral infections and cyclosporine related toxicity. Cyclosporine induced neurotoxicity is a well documented complication in stem cell transplant recipients. These patients usually present with seizures which are easily co...

Reactivation of Betapolyomavirus hominis (BKPyV) in kidney and hematopoietic stem cell recipients can lead to serious complications such as BKPyV-associated nephropathy followed by transplant rejection hemorrhagic cystitis. Early diagnosis the disease is hampering possible combination infection BKPyV with other post-transplant pathologies absence specific symptoms. Replication currently only re...

Fifty-five years after publication of the first hematopoietic stem cell transplantation this technique has become an accepted treatment option for defined hematologic and non-hematologic disorders. There is considerable interest in understanding differences in its use and trends on a global level and the macro-economic factors associated with these differences. Data on the numbers of hematopoie...

Tacrolimus, a potent immunosuppressant medication, acts by inhibiting calcineurin, which eventually leads to inhibition of T-cell activation. The drug is commonly used to prevent graft rejection in solid organ transplant and graft-versus-host disease in hematopoietic stem cell transplant patients. Tacrolimus has a narrow therapeutic index with variable oral bioavailability and metabolism via cy...

Viral infections are still a serious diagnostic and therapeutic problem in patients undergoing alternative donor transplants. β-Herpesviruses (especially human herpesvirus type 5, 6 and 7) are recognized pathogens in this group of patients and may cause central nervous system disease. Guillain-Barré syndrome (GBS) is a very rare complication among stem cell transplant recipients and usually has...

This paper reviews eight published studies of children who required critical care following a stem cell transplant. Approximately 14% of children required mechanical ventilation following stem cell transplant. Sixteen percent of these children survived. Eleven percent of children who had primary lung injury secondary to either infectious or non-infectious causes survived. Patients with respirat...

BACKGROUND We conducted a randomized trial of the treatment of multiple myeloma with high-dose chemotherapy followed by either one or two successive autologous stem-cell transplantations. METHODS At the time of diagnosis, 399 previously untreated patients under the age of 60 years were randomly assigned to receive a single or double transplant. RESULTS A complete or a very good partial resp...

OBJECTIVE This article analyzes sickle cell patient families' responses to stem cell transplant recruitment efforts. It identifies key dynamics that explain why sickle cell patient families are not undergoing stem cell transplants at the rate of other patient populations. It challenges the conventional focus on 'African-American distrust' as a set of attitudes grounded in collective memories of...