Pharming Recieves Orphan Drug Designations For rhC1INH From US FDA

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http://www.pharming.com/index.php?act=show&pg=278

Pharming Recieves Orphan Drug Designations For rhC1INH From US FDA

Leiden, The Netherlands, June 14, 2006. Biotech company Pharming Group
NV (“Pharming” or “the Company”) (Euronext: PHARM) announced today it
has received orphan drug designations for recombinant human C1
inhibitor (rhC1INH) from the Food and Drug Administration (FDA). The
Company has obtained designations on rhC1INH for two separate disease
indications – the prevention and/or the treatment of Delayed Graft
Function (DGF) after solid organ transplantation and the treatment of
Capillary Leakage Syndrome (CLS).

Over 25,000 solid organs were transplanted in the US in 2005, including
kidney, liver, lung and heart transplants. Delayed Graft Function is a
common complication affecting all solid organs in the post-transplant
period. DGF results in significant morbidity and mortality from early
graft dysfunction and from decreased long-term graft survival. The
condition also prolongs hospitalization and requires substitute
therapies for these patients, such as dialysis or ventilatory support.
DGF remains a critical unmet medical need despite improvements in
immunosuppression, organ preservation, and surgical technique. C1
inhibitor has been shown in numerous models of organ transplantation to
improve early graft function.

Over 100,000 patients in the US develop Capillary Leakage Syndrome
annually as a complication of various disease states, including bone
marrow/stem cell transplantation, IL-2 therapy, sepsis, and neonatal
cardiac surgery. CLS is a severe life-threatening condition
characterized by excessive fluid loss into the tissue space, which can
result in hemodynamic instability, pulmonary edema, ascites, and death.
Current therapies for patients with CLS are limited to supportive care
and treatment of the underlying condition. Previous clinical work has
demonstrated that C1 inhibitor may be an effective anti-inflammatory
that can control the mechanisms contributing to CLS.

“Pharming’s rhC1INH product could provide new treatments for immune
mediated diseases such as Delayed Graft Function in organ
transplantation and Capillary Leakage Syndrome, conditions with a high
burden and limited treatment options for patients,” said Dr. Francis
Pinto, CEO of Pharming. “The Orphan Drug designations from the FDA
further validate the potential of rhC1INH as an innovative therapy and
are a significant achievement as we advance development of rhC1INH for
these indications.”

The FDA Orphan Drug designation is reserved for promising new therapies
being developed to treat diseases that affect fewer than 200,000 people
in the United States. This designation provides an accelerated review
process, tax advantages and a seven-year period of market exclusivity
in the US upon product approval. Pharming also has an Orphan Drug
designation on rhC1INH for the treatment of Hereditary Angioedema.

Background on Pharming Group NV
Pharming Group NV is developing innovative protein products for unmet
needs. The Company’s products include potential treatments for genetic
disorders, specialty products for surgical indications, intermediates
for various applications and food products. Pharming has two products
in late stage development – recombinant human C1 inhibitor for
Hereditary Angioedema (Phase III) and recombinant human lactoferrin for
use in functional foods. The advanced technologies of the Company
include innovative platforms for the production of protein
therapeutics, as well as technology and processes for the purification
and formulation of these products. Additional information is available
on the Pharming website: http://www.pharming.com.

This press release contains forward looking statements that involve
known and unknown risks, uncertainties and other factors, which may
cause the actual results, performance or achievements of the Company to
be materially different from the results, performance or achievements
expressed or implied by these forward looking statements. The press
release also appears in Dutch. In the event of any inconsistency, the
English version will prevail over the Dutch version.

Contact:

Carina Hamaker, Investor Voice, T: +31 (0)6 537 49959
Sarah MacLeod, Financial Dynamics, T: +44 (0)20 7269 7148
Samir Singh, Pharming Group NV, T: +31 (0)71 524 7429

FDA Approves New Treatment for Myelodysplastic Syndromes (MDS), a Potential Secondary Disease to Treatment of Ewing's Sarcoma

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http://www.fda.gov/bbs/topics/NEWS/2006/NEW01366.html

FDA News

FOR IMMEDIATE RELEASE
P06-64
May 3, 2006
   

Media Inquiries:
Laura Alvey, 301-827-6242
Consumer Inquiries:
888-INFO-FDA

FDA Approves New Treatment for Myelodysplastic Syndromes (MDS)

The Food and Drug Administration (FDA) today approved Dacogen
(decitabine) injection for the treatment of myelodysplastic syndromes
(MDS). Dacogen is a new molecular entity that received orphan drug
status. Orphan products are developed to treat rare diseases or
conditions that affect fewer than 200,000 people in the U.S. The Orphan
Drug Act provides a seven-year period of exclusive marketing to the
first sponsor who obtains marketing approval for a designated orphan
drug.

Patients with MDS have bone marrow that does not produce enough mature
blood cells. This causes a lack of healthy blood cells that can
function properly in the body. Dacogen is thought to work by promoting
normal development of blood cells. Different types of MDS exist,
resulting in different manifestations of the disease. For example, some
patients with MDS require chronic blood transfusions.

“Today's approval of Dacogen offers patients with this rare disease an
additional treatment option that may help these patients avoid blood
transfusions,” said Steven Galson, MD, Director of FDA's Center for
Drug Evaluation and Research.

MDS can develop following treatment with drugs or radiation therapy for
other diseases or it can develop without any known cause. Some forms of
MDS can progress to acute myeloid leukemia (AML), a type of cancer in
which too many white blood cells are made.

An estimated 7,000 to 12,000 new cases of MDS are diagnosed yearly in
the United States. Although MDS occurs in all age groups, the highest
prevalence is in people over 60 years of age. Typical symptoms include
weakness, fatigue, infections, easy bruising, bleeding, and fever.

The safety and effectiveness of Dacogen were demonstrated in a
randomized, controlled trial where patients received either Dacogen or
the standard therapy and in two non-randomized studies where all of the
patients received Dacogen. The new drug was evaluated in a total of 268
patients. About 22% of patients in the three trials had complete or
partial responses to Dacogen. Responses consisted of complete or
partial normalization of blood counts and of fewer immature cells in
the bone marrow. In responders the need for transfusions was eliminated
during the period of response.

The most common side effects reported in clinical trials included
neutropenia (low white blood cell count), thrombocytopenia (low
platelets in blood), anemia, fatigue, fever, nausea, cough, bleeding in
the skin, constipation, diarrhea, and hyperglycemia (high blood sugar).

Dacogen is manufactured by Pharmachemie B.V. Haarlem, The Netherlands
for MGI PHARMA, INC., Bloomington, MN.

####

Risk of cancer in children with the diagnosis immaturity at birth

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http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=pubmed&dopt=Abstract&list_uids=16629697

Risk of cancer in children with the diagnosis immaturity at birth

Authors: Mellemkjær, Lene; Hasle, Henrik1; Gridley, Gloria2; Johansen,
Christoffer3; Kjær, Susanne K.3; Frederiksen, Kirsten3; Olsen, Jørgen
H.3

Source: Paediatric & Perinatal Epidemiology, Volume 20, Number 3,
May 2006, pp. 231-237(7)

Publisher: Blackwell Publishing

Abstract:
Summary Mellemkjær L, Hasle H, Gridley G, Johansen C, Kjær SK,
Frederiksen K, Olsen JH. Risk of cancer in children with the diagnosis
immaturity at birth. Paediatric and Perinatal Epidemiology 2006; 20:
231–237.

Cancer risk in children born before term has been assessed in a large
number of case–control studies but very rarely in cohort studies. We
carried out a cohort study of 35 178 children with the diagnosis
immaturity at birth in the Hospital Discharge Register during 1977–89.
The children were followed for cancer in the Danish Cancer Registry
until 1994 and comparisons were made with incidence rates for all
children in Denmark. The 64 observed cases of childhood cancer in the
cohort corresponded closely to the expected number {standardised
incidence ratio (SIR) = 1.03; [95% confidence interval (CI) 0.80,
1.32]}. The only cancer site with an observed number that deviated
significantly from the expected number was central nervous system (CNS)
tumours (26 cases observed; SIR = 1.57; [95% CI 1.02, 2.30]) in
particular medulloblastoma (9 cases observed; SIR = 3.1; [95% CI 1.4,
5.9]). In a nested case–control study of the CNS tumours, we found that
more cases than controls had been exposed to diagnostic X-rays, but the
result was not significant. Surprisingly, for those born before term,
the risk of CNS tumours increased with increasing gestational age in
the nested case–control data. Our results are in line with previous
evidence that children born before term are not at increased risk for
childhood cancer in general. An explanation behind the excess of CNS
tumours could not be identified, but the effect of diagnostic X-rays in
newborns may deserve further attention.

Keywords:  childhood cancer; CNS tumours; preterm; newborn X-rays

Document Type: Research article

DOI: 10.1111/j.1365-3016.2006.00717.x

Affiliations: 1: Department of Paediatrics, Aarhus University Hospital
Skejby, Aarhus, Denmark, and 2: Biostatistics Branch, Division of
Cancer Epidemiology and Genetics, National Cancer Institute, Rockville,
MD, USA 3: Institute of Cancer Epidemiology, Danish Cancer Society,
Copenhagen,

The Limb Preservation Foundation

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http://comment.colostate.edu/index.asp?page=display_article&article_id=396523010
The Limb Preservation Foundation
CBS News Program TONIGHT at 5:30 p.m.
The CBS Evening News will feature a story on today's broadcast (April 18
at 5:30 MST) about the collaborative efforts between Dr. Ross Wilkins of
the Denver Clinic for Extremities at Risk, and Dr. Stephen Withrow of
the Colorado State University Animal Cancer Center, and their efforts to
find the cause and the cure for bone cancer and other extremity diseases.
The April 18 news story will be entitled "A Tale of Two Doctors" and
will feature interviews with both doctors and information on their
research collaborations in regard to osteosarcoma. Drs. Wilkins and
Withrow have been working together for many years on collaborative
research pertaining to osteosarcoma treatments and protocols for
patients with limb cancer.
Thanks to their joint efforts, the survival rate of pediatric limb
cancer patients has increased from 60% in 1986 to 92% today. The Limb
Preservation Foundation is committed to the research being conducted by
these two doctors and is working to continue their efforts through the
development of a Limb Preservation University Chair in support of
research in Musculoskeletal Biology at the CSU Animal Cancer Center in
Fort Collins. The Foundation will be working with CSU to jointly fund
this research position with the goal of raising $3,000,000 together in
the next three years.
Dr. Ross Wilkins is Founder of The Limb Preservation Foundation and
Medical Director of the Denver Clinic for Extremities at Risk.
Dr. Stephen Withrow is the Director of the CSU Animal Cancer Center and
holds a seat on the Board of Directors of The Limb Preservation
Foundation. The Limb Preservation Foundation is a charitable
organization that was established in 1987. The mission of the Foundation
is to support the prevention and treatment of limb threatening
conditions due to trauma, tumor or infection.
The Foundation coordinates and funds patient treatment programs,
educational programs and research with the goal of addressing the needs
of individuals who are facing the potential loss of a limb.
Category: Events and Coming Attractions
Submitted: Tuesday, April 18, 2006
Subject: ACC
Contact: Lynda J Reed
E-mail: Lynda.Reed@colostate.edu
Phone: (970) 297-4175

Neurosurgical Use of Interstitial Laser Therapy (ILT)

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http://www.clinicaltrials.gov/ct/show/NCT00207350

Neurosurgical Use of Interstitial Laser
Therapy (ILT)

This study is currently
recruiting patients.
Verified by Brigham and Women's Hospital September 2005

Sponsored by: Brigham and Women's Hospital
Information provided by: Brigham and Women's Hospital
ClinicalTrials.gov Identifier: NCT00207350

Purpose

Our specific aims are
to test the following hypotheses: Hypothesis 1: A tumor can be
completely ablated by ILT with MRI-guidance; Hypothesis 2: The
MRI-based 3D temperature map of tissue during ILT is predictive of
destruction; Hypothesis 3: The 3D “thermal dose” map that is based on
the tissue’s temperature over time is more predictive of tissue
destruction than the temperature map.

Condition Intervention
Brain Tumor  Device: Interstitial Laser Therapy

MedlinePlus related
topics:  Brain Cancer

Study Type: Interventional
Study Design: Treatment, Non-Randomized, Open Label, Uncontrolled,
Single Group Assignment, Efficacy Study

Further study details as provided by Brigham and
Women's Hospital:
Primary Outcomes: A tumor can be completely
ablated by ILT with MRI-guidance
Secondary
Outcomes: Patients undergoing ILT will be assessed pre- and
post-operatively based on a neurological exam by a physician and
patient self-assessment using the Glioma Outcomes Questionnaire.
Expected Total Enrollment:  24

Study start: January 2002
Last follow-up: December 2004

The
goal is to evaluate the use of minimally invasive interstitial laser
therapy (ILT) in the brain. Our group is in a unique position to offer
image-guided ILT because of our expertise and resources here at Brigham
& Women's Hospital in the Departments of Neurosurgery and
Radiology. The therapy will be monitored and controlled by the use of
magnetic resonance imaging (MRI). ILT is a minimally invasive procedure
in which the targeted tissue is thermally destroyed in situ in a
controlled fashion. The intra-operative MRI provides a way to “see” the
treatment. It can be used to treat disease by guiding surgery by
providing images of tissue changes during therapy.

In spite of its appeal as a minimally
invasive technique, MRI-guided ILT is not commonly practiced in the
United States. One reason is that proper clinical implementation of ILT
requires an operating room (OR) setting and an MRI scanner – a very
rare combination. Our MRI-OR suite includes a sterile procedure room
with a 0.5 Tesla vertically “open” magnet. In the past, we have
performed MRI-guided ILT procedures in 9 patients. While few in number,
this is the most extensive U.S. experience in ILT in the brain.

We have recently created a new image
networking and display package for the visualization of 3D information
during laser therapy. This provides a view of multiple image planes
taken through the tissue volume around the fiber tip.

Each patient will undergo ILT. The
procedure will be performed under anesthesia as per standard
procedures. The surgical placement of the laser fiber is a procedure
identical to the well-developed and practiced technique of brain
biopsy. A hole approximately 1 cm in diameter will be drilled in the
skull through which the laser fiber will be placed under image guidance
to confirm the actual progress during the advance of the fiber. We will
deliver energy at a rate and distribution of 1-12 watts/cm for
exposures less than 20 minutes. After the laser has been turned off,
and the tissue cooled, MRI will show the region of ablation. As needed,
the laser fiber will be moved/re-located to assure that the total
target has been ablated. After the treatment is complete, the fiber is
withdrawn, final images are acquired and the surgical site is closed
and dressed. On the day after the procedure, the patient will undergo a
24 hr follow-up MRI exam. There will be post-operative care as with any
neurosurgical patient.

The following continuous variables will be measured in this study.

  • the pre-operative tumor volume (VO) in cc
  • the post-operative ablated volume (V1) in cc
  • the intra-operative critical temperature volume (VT) in cc
  • the intra-operative critical dose
    volume (VD) in cc.

The following statistical hypothesis tests will be conducted.

Statistical Hypothesis 1. A tumor can be completely ablated by ILT
with MRI-guidance.

We
propose that the difference between the mean the pre-op tumor volumes
and the post-op ablated volumes (VO and V1, respectively) is zero.
Residual tumor is defined as (V0-V1). This will be determined by
calculating the mean of the values of the proportion of residual tumor,
defined as (V0-V1)/ V0.Use of the proportion normalizes the data for
different sized tumors.

Statistical Hypothesis 2. The MRI-based
3-D temperature map of the tissue during ILT is predictive of
destruction. We propose that the difference between the mean post-op
ablated volumes and the intra-operative critical temperature volumes
(VT and V1, respectively) is zero. This will be determined by
calculating the mean of the values of the proportion of the difference
between them, defined as (VT-V1)/VT.

Statistical Hypothesis 3. The thermal dose map is predictive of
tissue destruction.

We
propose that the difference between the mean post-op ablated volumes
and the intra-operative critical dose volumes (VD and V1, respectively)
is zero. This will be determined by calculating the mean of the values
of the proportion of the difference between them, defined as (VD-V1
/VD).

Also, data will be collected through Neurological Examinations and
GOC Questionnaire.

Eligibility

Ages Eligible for
Study:  18 Years and above,  Genders Eligible for Study:  Both
Criteria

Inclusion Criteria:

Male or female Age 18+ Surgically difficult to access tumors
including intracerebral metastases and vascular malformations

–

Exclusion Criteria:

Patients
unwilling or unable to give written consent Patients at risk for
cardiac ischemia Patients who cannot physically fit in the MRI scanner
in the MRI OR Patients with contra-indications to MRI imaging such as
pacemakers, non-compatible aneurysm clips, shrapnel, and other internal
ferromagnetic objects Patients with coagulopathies, severe medical
problems, cardiac arrythmias or abnormal BUN –

Location and Contact Information

Please refer to this study by ClinicalTrials.gov
identifier  NCT00207350
Joanne E O'Hara, M.A.      617-732-6992    johara1@partners.org
Donna Dello Iacono, RN, MS      617-732-6826    ddelloiacono@partners.org
Massachusetts
      Brigham & Women's Hospital, Boston,  Massachusetts,  02115, 
United States; Recruiting

Peter M Black, MD, PhD,  Principal Investigator

Study chairs or principal investigators
Peter M Black, MD, PhD,  Principal Investigator,  Brigham and Women's
Hospital   

More Information

Website
for Image Guided Therapy Program at Brigham & Women's Hospital

Publications

Schulze
PC, Vitzthum HE, Goldammer A, Schneider JP, Schober R. Laser-induced
thermotherapy of neoplastic lesions in the brain–underlying tissue
alterations, MRI-monitoring and clinical applicability. Acta Neurochir
(Wien). 2004 Aug;146(8):803-12. Epub 2004 Jun 7. Review.

Peller
M, Muacevic A, Reinl H, Sroka R, Abdel-Rahman S, Issels R, Reiser MF.
[MRI-assisted thermometry for regional hyperthermia and interstitial
laser thermotherapy] Radiologe. 2004 Apr;44(4):310-9. German.

McNichols
RJ, Gowda A, Kangasniemi M, Bankson JA, Price RE, Hazle JD. MR
thermometry-based feedback control of laser interstitial thermal
therapy at 980 nm. Lasers Surg Med. 2004;34(1):48-55.

Leonardi
MA, Lumenta CB. Stereotactic guided laser-induced interstitial
thermotherapy (SLITT) in gliomas with intraoperative morphologic
monitoring in an open MR: clinical expierence. Minim Invasive
Neurosurg. 2002 Dec;45(4):201-7.

Straube
T, Kahn T. Thermal therapies in interventional MR imaging. Laser.
Neuroimaging Clin N Am. 2001 Nov;11(4):749-57. Review.

Muacevic
A, Peller M, Sroka R, Kalusche B, Pongratz T, Kreth FW, Steiger HJ,
Reiser M, Reulen HJ. [Brain protective interstitial laser
thermotherapy. Therapy of brain tumors without secondary damage] MMW
Fortschr Med. 2001 May 28;143 Suppl 2:87-8. German.

Study ID Numbers:  2001-P-001794
Last Updated:  December 8, 2005
Record first received:  September 12, 2005
ClinicalTrials.gov Identifier:  NCT00207350
Health Authority: United States: Institutional Review Board
ClinicalTrials.gov processed this
record on 2006-04-18

Pediatric Cancers Are Infiltrated Predominantly by Macrophages and Contain a Paucity of Dendritic Cells: a Major Nosologic Difference with Adult Tumors

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Clinical Cancer Research Vol. 12, 2049-2054, April 2006
© 2006 American Association for Cancer Research
Human Cancer Biology

Pediatric Cancers Are Infiltrated Predominantly by Macrophages and
Contain a Paucity of Dendritic Cells: a Major Nosologic Difference with
Adult Tumors
Jukka Vakkila1,2, Ronald Jaffe3, Marilyn Michelow3 and Michael T. Lotze1

http://clincancerres.aacrjournals.org/cgi/content/abstract/12/7/2049

Authors' Affiliations: 1 Molecular Medicine Institute, University of
Pittsburgh School of Medicine, Pittsburgh, Pennsylvania; 2 Department
of Bacteriology and Immunology, Haartman Institute, University of
Helsinki, Helsinki, Finland; and 3 Children's Hospital of Pittsburgh,
University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania

Requests for reprints: Jukka Vakkila, Department of Bacteriology and
Immunology, Haartman Institute, Haartmaninkatu 31, FIN-00014 University
of Helsinki, Helsinki, Finland. Phone: 358-40-821-6939; Fax:
358-10-414-4619; E-mail: jukka.vakkila@mehilainen.fi.

Purpose: Adult cancer is frequently preceded by a period of prolonged
chronic inflammation caused by infectious microbial agents or physical
or chemical irritants. By contrast, an association between the classic
pediatric neoplasias and inflammatory triggers is only rarely
recognized. We hypothesized that the difference could be reflected in
the inflammatory cell infiltrates of pediatric and adult cancer.

Experimental Design: Three investigators retrospectively studied 27
pediatric and 13 adult cancers at first diagnosis by
immunohistochemistry. Inflammatory cells were identified and counted,
and their location in relation to tumor tissue was analyzed.

Results: A majority of tumor-associated leukocytes (TAL) in adult
tumors were located at the edges of tumor islands forming inflammatory
foci between the supporting stroma and the malignant infiltrate. In
contrast, TALs in pediatric tumors were scattered within the malignant
tumor islands. In adult tumors, TALs were composed of diverse leukocyte
types; but in pediatric tumors, the infiltrating cells were
predominantly macrophages that accumulated in areas of necrosis within
the tumors. The most striking feature in the pediatric tumors was the
virtual absence of dendritic cells. The proportion of intratumoral
dendritic cells in pediatric samples was 4.1%; whereas in adult tumors,
they formed 36.9% of TALs within the tumor islands and 25.1% around the
tumors.

Conclusions: We conclude that TALs in pediatric cancers are composed
mainly of macrophages and largely devoid of dendritic cell. The
findings may provide a major nosologic difference reclassifying
pediatric and adult tumors based on nominal inflammatory and
noninflammatory etiologies.

High histologic and overall response to dose intensification of ifosfamide, carboplatin, and etoposide with cyclophosphamide, doxorubicin, and vincristine in patients with high-risk ewing sarcoma family tumors

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http://www3.interscience.wiley.com/cgi-bin/abstract/112476870

High histologic and overall response to dose intensification of
ifosfamide, carboplatin, and etoposide with cyclophosphamide,
doxorubicin, and vincristine in patients with high-risk ewing sarcoma
family tumors

The Bambino Gesù Children's Hospital experience
Giuseppe Maria Milano, M.D. 1 *, Raffaele Cozza, M.D. 1, Ilaria Ilari,
M.D. 1, Luigi De Sio, M.D. 1, Renata Boldrini, M.D. 2, Alessandro
Jenkner, M.D. 1, Maretta De Ioris, M.D. 1, Alessandro Inserra, M.D. 3,
Carlo Dominici, M.D. 1 4, Alberto Donfrancesco, M.D. 1
1Division of Pediatric Oncology, Ospedale Pediatrico Bambino
Gesù-IRCCS, Rome, Italy
2Division of Pathology, Ospedale Pediatrico Bambino Gesù-IRCCS, Rome,
Italy
3Division of Pediatric Surgery, Ospedale Pediatrico Bambino Gesù-IRCCS,
Rome, Italy
4Department of Pediatrics, La Sapienza University, Rome, Italy
email: Giuseppe Maria Milano (milano_gm@hotmail.com)

*Correspondence to Giuseppe Maria Milano, Dipartimento di
Oncoematologia Pediatrica e Servizio Immunotrasfusionale, UO di
Oncologia, Ospedale Pediatrico Bambino Gesù-IRCCS, Piazza Sant'Onofrio
4, Rome, 00165, Italy
Fax: (011) 396 68592242

Keywords
Ewing sarcoma family tumors • chemotherapy • dose intensification •
histologic response • tumor necrosis • survival

Abstract

BACKGROUND
Ewing sarcoma (ES) and extraosseous ES/primitive neuroectodermal tumors
(PNET) share histopathologic features of the ES family of tumors
(ESFT). The authors report on their results from a regimen of
ifosfamide, carboplatin, and etoposide (ICE) with cyclophosphamide,
doxorubicin, and vincristine (CAV) dose intensification in patients
with high-risk ESFT.

METHODS
Since 1990, patients with ESFT and with 1 or more of the following risk
factors were reviewed: tumor volume > 200 mL, tumor site with a poor
prognosis, and pulmonary and/or bone marrow metastases.

RESULTS
Thirty-six patients with ESFT who were involved in the study were
divided into 2 arms of 18 patients each. One group received treatment
with various regimens, and the other group received treatment with ICE
plus CAV. The disease was brought under control more rapidly in the
latter patients, for whom surgery was more easily feasible, and up to
90% of patients achieved a major response, with an estimated 3-year
overall survival rate of 67% ± 12%.

CONCLUSIONS
The current results showed that ICE plus CAV was tolerated well and was
effective in the studied subset of tumors, indicating that dose
intensification correlates with better disease control, a high
percentage of necrosis, and conservative surgery in patients with
high-risk ESFT. Cancer 2006. © 2006 American Cancer Society.

ALK-positive anaplastic large cell lymphoma with primary bone involvement in children.

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Am J Clin Pathol. 2006 Jan;125(1):57-63. Related Articles, Links 

ALK-positive anaplastic large cell lymphoma with primary bone
involvement in children.

NA, Ross CW, Finn WG, Valdez R, Ruiz R, Koujok K, Schnitzer B.

http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=16482992&dopt=Abstract

Department of Pathology, Oklahoma University Health Sciences Center,
Oklahoma City, USA.

We describe the clinical, radiologic, and pathologic features of
primary bone anaplastic large cell lymphoma (ALCL) in 3 boys.
Radiologic imaging showed lytic lesions involving sacrum, femur, or
rib. Bone was the only site of disease in 2 cases; an associated
partial lymph node was involved in case 3. Differential diagnoses
included osteomyelitis and small round cell tumors of childhood,
particularly Ewing sarcoma. Preoperatively, ALCL was not a diagnostic
consideration in any case. Two cases showed classic large pleomorphic
cells; 1 showed a composite pattern with a distinct small cell
component and the more typical large cell type. Neoplastic cells in all
cases showed strong CD30 and anaplastic lymphoma kinase expression with
relatively weak epithelial membrane antigen positivity. Cytotoxic
granule protein was expressed in 2 cases. All cases showed unusually
strong expression of neuron-specific enolase (NSE). Two patients were
disease-free at last follow-up (15 months and 11 years); 1 patient died
of disseminated disease within a year of diagnosis. ALCL should be
considered a diagnostic possibility when evaluating neoplastic bone
lesions in children. Although expression of NSE in ALCL has not been
emphasized in the literature, it is worth noting because it may pose a
diagnostic pitfall.

PMID: 16482992 [PubMed – in process]

Ewing's sarcoma of bone: the detection of specific transcripts in a large, consecutive series of formalin-fixed, decalcified, paraffin-embedded tissue samples using the reverse transcriptase-polymerase chain reaction.

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Histopathology. 2006 Mar;48(4):363-76.
 
Ewing's sarcoma of bone: the detection of specific transcripts
in a large, consecutive series of formalin-fixed, decalcified,
paraffin-embedded tissue samples using the reverse
transcriptase-polymerase chain reaction.

Mangham DC, Williams A, McMullan DJ, McClure J, Sumathi VP, Grimer RJ,
Davies AM.

http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=16487358&dopt=Abstract

Department of Musculoskeletal Pathology, Royal Orthopaedic Hospital,
The Medical School, University of Birmingham, Birmingham, UK.

Aims : (i) To report on the routine use of the reverse
transcriptase-polymerase chain reaction (RT-PCR) technique on
decalcified or non-decalcified, formalin-fixed, paraffin-embedded
tissue (FFPET) for translocation detection, with particular emphasis on
improved RNA extraction methodology and the use of PCR primers designed
to generate small amplicons. (ii) To report on the relative incidences
of translocation types and transcript variants in a large, single
institution series of Ewing's sarcoma of bone. Methods and results :
Using RT-PCR to detect specific transcript variants, we analysed FFPET
from 54 consecutive cases of Ewing's sarcoma of bone. We used 'gold
standard' detection methods on corresponding fresh and fresh frozen
tissue to validate the technique. We have demonstrated the effective
use of RT-PCR on decalcified and non-decalcified FFPET samples for
sarcoma-specific translocation detection (96% sensitivity, 100%
specificity). Tissue decalcification did not affect the detection rate.
The relative incidence of Ewing's sarcoma-specific translocation types
and transcript variants was entirely consistent with previously
published data. Conclusions : With equal effectiveness, RT-PCR can be
applied to both acid decalcified and non-decalcified FFPET for (Ewing's
sarcoma) translocation detection and the technique can be introduced
into routine practice in histopathology departments.

PMID: 16487358 [PubMed – in process]

Laparoscopic Removal of Extraosseous Ewing's Sarcoma of the Kidney in a Pediatric Patient.

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J Laparoendosc Adv Surg Tech A. 2006 Feb;16(1):74-6.

Laparoscopic Removal of Extraosseous Ewing's Sarcoma of the
Kidney in a Pediatric Patient.

http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=16494555&dopt=Abstract
 

Perer E, Shanberg AM, Matsunaga G, Finklestein JZ.

Miller Children's Hospital, Long Beach Memorial Medical Center, Long
Beach, California., Antoci Center for Pediatric Urology and Nephrology,
UC Irvine Medical Center, Orange, California.

In the pediatric population, to the best of our knowledge, only 2 cases
of renal extraosseous Ewing's sarcoma/primitive neuroectodermal tumor
(EES/PNET) have been published. We report the initial case of renal
EES/PNET occurring in a 10-year-old girl treated by a laparoscopic
radical nephrectomy. The regimen used is the first documented use of
neoadjuvant chemotherapy prior to laparoscopic radical nephrectomy for
PNET. This approach obviated the need for a large incision and a
prolonged postsurgical recovery. The minimally invasive nature of the
laparoscopic procedure allowed for a rapid convalescence and resumption
of her chemotherapy regimen within 14 days of the surgery.

PMID: 16494555 [PubMed – in process]