Prospective assessment of health-related quality of life in pediatric patients with beta-thalassemia following hematopoietic stem cell transplantation.

Although hematopoietic stem cell transplantation (HSCT) has been widely used to treat pediatric patients with beta-thalassemia major, evidence showing whether this treatment improves health-related quality of life (HRQoL) is lacking. We used child-self and parent-proxy reports to prospectively evaluate HRQoL in 28 children with beta-thalassemia from Middle Eastern countries who underwent allogeneic HSCT in Italy. The PedsQL 4.

Epigenetic alterations of p15(INK4B) and p16(INK4A) genes in pediatric primary myelodysplastic syndrome.

We studied the methylation status of the p15(INK4B) and p16(INK4A) genes in 47 pediatric patients with primary MDS, its correlation with subtype, and the role of p15(INK4B) and p16(INK4A) in the evolution of MDS toward AML. Aberrant methylation of the p15(INK4B) gene was detected in 15 of 47 patients (32%), whereas only four patients demonstrated methylation of the p16(INK4A) gene (8%). The frequency of p15(INK4B) methylation was significantly higher in RAEB and RAEB-t subtypes (p<0.

Epididymo-orchitis and central nervous system nocardiosis in a bone marrow transplant recipient for acute lymphoblastic leukemia.

Objectives: We report a case of epididymo-orchitis and central nervous system nocardiosis in a 22-year-old man with T-cell acute lymphoblastic leukemia; he was an allogeneic marrow recipient with acute and chronic graft-versus-host disease.

Materials And Methods: He had microscopic hematuria and cytomegalovirus antigenemia. He deteriorated subsequently while on cyclosporine and steroids, requiring hospital admission owing to fever and swelling of the left testis and generalized tonic-clonic convulsions.

Antiproliferative effect of rapamycin on human T-cell leukemia cell line Jurkat by cell cycle arrest and telomerase inhibition.

Aim: To examine the ability of rapamycin to suppress growth and regulate telomerase activity in the human T-cell leukemia cell line Jurkat.

Methods: Cell proliferation was assessed after exposure to rapamycin by 3-(4,5-dimethylthiazol- 2-yl)-2,5-diphenyltetrazolium bromide assay. Cell cycle progression and apoptosis were determined by flow cytometry.

Allogeneic stem cell transplantation in hematological disorders: single center experience from Pakistan.

One hundred and fifty-four patients received allogeneic stem cell transplantations from HLA-matched siblings for various hematological disorders from July 2001 to September 2006. Indications for transplantation included aplastic anemia (n=66), beta-thalassemia major (n=40), CML (n=33), acute leukemia (n=8), and miscellaneous disorders (n=7). One hundred and twenty patients were males and 34 were females.

Cytogenetic biclonality in a child with hypocellular primary myelodysplastic syndrome.

A 13-year-old boy with hypocellular primary myelodysplastic syndrome, classified as refractory cytopenia, underwent umbilical cord blood transplantation. Cytogenetic analysis revealed two rare biclonal chromosomal aberrations, del(17)(p12) and del(11)(q23). Cytogenetic analysis was a valuable tool in diagnosis, in clinical decision-making, and in treatment and follow-up.

[Treatment of childhood leukemia with unrelated donor allogeneic bone marrow transplantation].

Objective: Allogeneic bone marrow transplantation has been established as a standard method for the treatment of a range of malignant and non-malignant hematologic diseases in children. Unfortunately, fewer than 30% of patients have a human leukocyte antigen (HLA)-matched sibling. Advances in our understanding of the HLA system and the development of large international donor registries encourage the increasing use of unrelated donors as an alternative source of stem cells.

Autologous serum for isolation and expansion of human mesenchymal stem cells for clinical use.

Objective: Mesenchymal stem cells (MSC) are promising candidates for cell-based therapies. One major obstacle for their clinical use is the biosafety of fetal calf serum (FCS), which is a crucial part of all media currently used for the culture of MSC.

Methods: Nine donors each contributed 5 mL of bone marrow aspirate.

[A comparison of clinical outcomes between HLA allele matched and 1 - 2 alleles mismatched unrelated allogeneic bone marrow transplantations].

Objective: To compare the clinical outcomes between HLA allele matched (HLA-M) and 1 approximately 2 alleles disparity mismatched (HLA-mis) unrelated allogeneic bone marrow transplantation (URD-BMT).

Methods: Thirty-nine patients received HLA-M and 21 received HLA-mis URD-BMT for the treatment of acute leukemia, chronic myeloid leukemia in chronic phase (CP) and myelodysplastic syndromes (MDS) in our hospital between November 1998 and December 2002. Conditioning regimen was Bu 16 mg/kg plus CTX 120 mg/kg, and mycophenolate mofetil (MMF), CsA and MTX were given to prevent aGVHD.

Hickman catheter embolism in a child during stem cell transplantation.

The majority of stem cell recipients rely on indwelling central venous catheters situated in superior vena cava or right atrium. Semi-permanent tunneled silicone rubber Hickman catheters are widely used to provide durable central venous access for patients undergoing stem cell transplantation. A case of 5 years old child with diagnosis of severe aplastic anemia is reported.

Human mesenchymal stem cells are not of donor origin in patients with severe aplastic anemia who underwent sex-mismatched allogeneic bone marrow transplant.

Stromal defects are part of the etiology of severe aplastic anemia (SAA), and hematopoietic engraftment is poor in unrelated and mismatched transplant. Therefore, we wanted to find out whether human mesenchymal stem cells (MSC) are partly of donor origin in patients with SAA years after successful bone marrow transplant (BMT). Three SAA patients 3, 5, and 8 years after BMT (cyclophosphamide, ATG) with bone marrow from an HLA-identical sibling donor of the opposite sex were investigated.

Allogeneic hematopoietic stem cell transplantation in the active duty military population: 1987-2001.

Wilford Hall Medical Center at Lackland Air Force Base is the only military medical center in the United States at which allogeneic hematopoietic stem cell transplants (HSCT) are performed. Through May 2001, 367 patients, including 163 active duty service members, have received allogeneic HSCT from related donors. We performed a retrospective review of the outcomes of allogeneic HSCT in active duty members to determine what proportion was returned to full-time military service after HSCT and to delineate the factors associated with successful return to military service.

Stem cell transplantation for thalassemia.

Thalassemia, one of the most common genetic disorders, is considered to be a global problem. Several millions of the patients suffer from severe thalassemic diseases. Stem cell transplantation is currently the only curative therapy.

Purification of human hemopoietic stem cells for transplantation in Thailand.

Stem cell transplantation (SCT) has become the therapy of choice for many hematologic and immunologic disorders. At present, only 25% of patients have suitable HLA-identical donors. In an attempt to increase the donor pool for SCT in Thailand and Southeast Asia, we developed a program whereby parents and mismatched siblings can be used as donors.

Acanthamoeba meningoencephalitis following autologous peripheral stem cell transplantation.

Amoebic meningoencephalitis is an unusual complication of bone marrow transplantation. We report a case of Acanthamoeba meningoencephalitis in a patient with non-Hodgkin's lymphoma after autologous stem cell transplantation. Leg weakness, fever and urinary retention developed 69 days following transplantation.

Umbilical cord blood transplants: treatment for selected hematologic and oncologic diseases.

Umbilical cord blood transplantation is a rapidly growing form of treatment for many types of cancer and hematologic disorders. The concepts behind the use of umbilical cord blood transplantation are based on information gained from experience in bone marrow transplantation. Previously discarded as human waste, the blood in the umbilical cord remnant and the placenta has been observed to be rich in hematopoietic stem cells.

Stomatococcus mucilaginosus meningitis in a patient with multiple myeloma following autologous stem cell transplantation.

Bacterial meningitis is an unusual complication of bone marrow transplantation. We report a case of Stomatococcus mucilaginosus meningitis in a patient with multiple myeloma shortly after an autologous peripheral blood stem cell transplant. The infection resolved with a combination of intravenous penicillin G and chloramphenicol, and intrathecal vancomycin.

An autonomously replicating eukaryotic expression vector with a tetracycline-responsive promoter.

The control elements of the tetracycline-resistance operon encoded in Tn10 of Escherichia coli have previously been utilized by Gossen and Bujard [ Proc. Natl. Acad.

Collection of cord blood stem cells for transplantation in thalassemic patients.

Thalassemia is widely distributed throughout the world and is one of the major public health problems. The use of bone marrow transplantation, the only curative therapy for thalassemia, is limited because less than 30% of the patients have unaffected and HLA-identical siblings as donors. Cord blood stem cells, an alternative source of stem cells for transplantation, have been successfully transplanted into patients with several diseases after myeloablative therapy.

Use of cryopreserved bone marrow in allogeneic bone marrow transplantation.

Use of cryopreserved donor bone marrow may facilitate scheduling of allogeneic bone marrow transplantation (BMT) by affording independence of a fixed time for bone marrow donation. The potential risk of damage to hematopoietic stem cells by cryopreservation resulting in delayed engraftment or graft failure has to be taken into account, however. To address these issues, the outcome of 19 matched related BMT (1992-94) performed with cryopreserved donor bone marrow was analyzed and compared with 19 related BMT (1990-93) receiving fresh donor bone marrow (control group).

Circulating tumor gangliosides enhance platelet activation.

Gangliosides enhance tumor formation in experimental animals, and high circulating concentrations of gangliosides shed by tumor cells are associated with rapid progression of human neuroblastoma. We studied these shed molecules for effects on platelet function, because platelet activation may play a role in the metastatic process. Preincubation of normal platelets in patient (tumor ganglioside-containing) serum resulted in their aggregation upon exposure to a subthreshold concentration (1 microgram/mL) of collagen (up to 34% v < 10% in normal serum) and ATP release (up to 1.

Cyclosporine monitoring improves graft-versus-host disease prophylaxis after bone marrow transplantation.

Objective: The principal objective of this study was to determine whether a relationship exists between trough cyclosporine concentrations measured by HPLC and the development of acute graft-versus-host disease (GVHD) after allogeneic bone marrow transplantation.

Design: A retrospective analysis of 59 consecutive human leukocyte antigen-matched bone marrow transplants. Patients received uniform GVHD prophylaxis with cyclosporine and methotrexate.

Prevention of hypermenorrhea with leuprolide in premenopausal women undergoing bone marrow transplantation.

We report on the use of leuprolide to prevent heavy menstrual bleeding that often occurs before platelet engraftment in premenopausal women undergoing bone marrow transplantation (BMT). Leuprolide, a synthetic analog of gonadotropin-releasing hormone (Gn-RH-a), was given to 34 patients by intravenous bolus injection, 1 mg daily, until platelet recovery. The median duration of therapy was 50 days (range 16-170).