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Abstract: Allogeneic hematopoeitic progenitor cell transplantation (HPCT) is a curative therapy for pediatric patients with both malignant and nonmalignant diseases. Single or multiple benign exostoses or osteochondromas have been reported after total body irradiation (TBI), as well as after focal irradiation. Patients exposed to TBI at a young age are at highest risk of developing exostoses. The objective of this institutional review board–approved study was to look at potential factors, besides radiation, that may play a role in development of exostoses. All patients who underwent allogeneic and autologous HPCT at a single institution between March 1992 and December 2003 and who developed an exostosis identified by clinical findings or as an incidental finding on a radiologic study were included. A case-control design matched patients with controls who had the same stem cell source. [Copyright &y& Elsevier]

Post-hematopoietic stem cell transplantation (hsCT) chimerism monitoring is important to assess relapse and therapeutic intervention. The purpose of our study is to compare two methods variable number tandem repeats (VNTR) vs. quantitative real- time polymerase chain reaction (qpCR) in terms of determining chimerism. 127 (peripheral blood n = 112, bone marrow n = 15) samples were simultaneously tested by VNTR using apO-B, D1s80, D1s111, D17s30, gene loci sRY and Zp3 and qpCR using 34 assays (Ca001-Ca034) that are designed to a bi-allelic insertion/deletion (indel) polymorphism in the human genome. samples were separated in three subsets: total WBC, T-cell and Myeloid cells. extraction of DNa was performed then quantified. We analyzed column statistics, paired t-test and regression analysis for both methods. There was complete correlation between the two methods. The simplicity and rapidity of the test results from the qpCR method is more efficient and accurate to assess chimerism. [ABSTRACT FROM AUTHOR]

Andolina JR, Kletzel M, Tse WT, Jacobsohn DA, Duerst RE, Schneiderman J, Helenowski I, Rademaker A, Chaudhury S. Allogeneic hematopoetic stem cell transplantation in pediatric myelodysplastic syndromes: Improved outcomes for de novo disease. Pediatr Transplantation 2011: 15: 334-343. © 2011 John Wiley & Sons A/S. We report 23 consecutive pediatric patients with MDS who received allogeneic HSCT on IRB approved protocols between 1992 and 2009 at Children's Memorial Hospital (Chicago, IL). Nine patients had de novo MDS, whereas 14 patients had treatment-related MDS. All patients had a documented cytogenetic abnormality, and monosomy 7/7q− was seen in 12 patients (52%). Fourteen of 23 patients received a myeloablative conditioning regimen; RIC regimens were used for the remaining nine. Five patients relapsed post-transplant, including four patients who received RIC transplant and four patients with treatment-related MDS. For the entire group, estimated five-yr RFS and OS were 47% and 50%, respectively. Treatment-related MDS was associated with decreased RFS in comparison with de novo MDS (33% vs. 70%, p = 0.05). Five-year OS rates reached 80% for those with de novo MDS. RIC regimens were associated with decreased three-yr RFS in comparison with myeloablative regimens (22% vs. 68%, p = 0.02). There was no correlation of survival with blast count at diagnosis, IPSS score, cytogenetic abnormality, donor type, or HLA match. Larger series are needed to confirm prognostic factors so that higher-risk patients can be targeted with novel approaches. [ABSTRACT FROM AUTHOR]

We describe the experience with reduced intensity transplants (RIT) in pediatric patients and review the basis for this approach using adult examples and series of patients. We report the experience with RIT in two pediatric studies. [ABSTRACT FROM AUTHOR]

The author discusses the manuscript regarding the treatment for high risk solid tumors in children having myeloablative chemotherapy and autologous hematopoietic progenitor stem cell (HPC) transplantation. He says that the true effect of autologous HPC in treatment cancer can be determined by performing randomize trials which compares the standard therapy to the use of high-dose therapy. He mentions that therapy and radiation received by patients should be considered.

The article focuses on a study regarding children with chronic myeloid leukemia (CML) and long term of effect of tyrosine kinase inhibitor (TKI) therapy and hematopoietic stem cell transplantation (HSCT). Topics discussed include significantly less number of percentage of CML in the pediatric population, introduction of the TKI imatinib by the U.S. in 1988, development of short term TKI toxicity, and improvement in overall survival of patients.

Background Transplantation of allogeneic hemopoietic stem cells can cure several non-malignant disorders in children. Transplantation with reduced intensity preparation might achieve the same goals but with less toxicity. We undertook a pilot study to determine engraftment rates, kinetics of engraftment, toxicity, and acute graft-versus-host disease (GVHD) associated with a uniform reduced intensity hemopoietic stem-cell transplant (HSCT) regimen for children with non-malignant diseases. Methods We studied 13 pediatric patients with non-malignant disorders who underwent reduced intensity HSCT at Children's Memorial Hospital from January, 2000, to February, 2004. Stem-cell sources included unrelated donor, matched-sibling peripheral blood stem cells, and unrelated cord blood. A uniform preparative regimen was used, consisting of fludarabine, busulfan, and anti-thymocyte globulin. Major endpoints were engraftment, transplant-related mortality at day 100, short-term toxicities, and incidence of acute GVHD. Results 72% of evaluable patients achieved full donor engraftment. There was rapid reconstitution of platelets (median 13.5 days) and neutrophils (median 18 days). Short-term toxicities were minimal, as seen by a median length of hospital stay of 7 days (between days 0-100). Incidence of grade II-IV acute GVHD was 8%. Two patients died before day 100 from underlying disease and viral infection, respectively (day 100 transplant-related mortality of 15%). The 1-year overall survival was 84% (95% CI 64-100). Most patients with immunodeficiencies and metabolic disorders had excellent donor engraftment and disease resolution or stabilization, but most of those with hemoglobinopathies rejected their graft. Interpretation This reduced intensity regimen followed by HSCT provides a good alternative to myeloablative HSCT for children with non-malignant disorders, except for hemoglobinopathies, in which engraftment is poor. [ABSTRACT FROM AUTHOR]

Conclusions. Nonmyeloablative HSCT from HLA-matched siblings can offer immune reconstitution with-out hepatotoxicity in patients with X-HIM syndrome and preexisting cholangiopathy. Even with stable mixed chimerism after allogeneic HSCT, patients may be able to enjoy a normal phenotype. Nonmyeloablative HSCT warrants additional study in X-HIM syndrome. Pediat-rics 2004;113:e122--e127. URL: http://www.pediatrics.org/ cgi/content/full/113/2/e122; stem cell transplant, nonmy-eloablative, immunodeficiency, hyper-IgM, cholangiopathy. [ABSTRACT FROM AUTHOR]