Featured Research

from universities, journals, and other organizations

Tailored doses of cytostatic improve survival rate after stem cell transplantation

Date:
October 23, 2013
Source:
Karolinska Institutet
Summary:
Researchers have managed to improve cytostatic therapy for children with the chronic immune deficiency disorder granulomatous disease prior to stem cell transplantation. By tailoring doses of the cytostatics administered before the transplantation, the researchers achieved a higher rate of survival with minimal adverse reactions. Now more patient groups are to undergo the same therapeutic strategy.

Researchers at Karolinska Institutet in Sweden, and colleagues at University Children's Hospital Zόrich in Switzerland have managed to improve cytostatic therapy for children with the chronic immune deficiency disorder granulomatous disease prior to stem cell transplantation. By tailoring doses of the cytostatics administered before the transplantation, the researchers achieved a higher rate of survival with minimal adverse reactions. Now more patient groups are to undergo the same therapeutic strategy.

The results, which are presented in the scientific journal The Lancet, are based on a clinical study conducted at 16 hospitals around the world. Chronic granulomatous disease (CGD) is a rare immune deficiency in children that causes recurrent, often difficult-to-treat bacterial and fungal infections and non-bacterial inflammations of the inner organs, which sometimes develop into tumour-like nodules of inflammatory tissue known as granuloma. By monitoring patients with this disease, the researchers conducting the study developed a protocol for optimising preparatory treatment with cytostatics (cancer drugs) prior to stem cell transplantation. Careful sampling allowed the doses to be adjusted and optimised for each patient, a procedure that resulted in much better survival rates and minimal adverse reactions.

"We can now present a survival rate of 93 per cent, and that with available cytostatics rather than a new drug," says research team member Moustapha Hassan, Professor of transplantation research at Karolinska Institutet's Department of Laboratory Medicine. "This is a terrific result! Particularly so given that it's babies that we can now help."

Stem cell transplantation is the only treatment method available today for CGD, and is usually preceded by a course of cytostatic drugs to help the body accept the transplantation. However, one problem is that excessive doses of cytostatics can harm the recipient's organs, leading to complications such as central nervous system damage and infertility, while insufficient doses can cause the patient's body to reject the transplanted stem cells.

"Babies are extremely sensitive to cytostatics, which makes it especially important to find the right dose for each individual patient," says Professor Hassan. "This we've managed to do by monitoring the concentration of cytostatics in their blood and adjusting the dose accordingly to obtain optimal levels for maximal effect and minimal toxicity."

The study involved pre-treating the patients with high doses of the cytostatic fludarabine combined with the immunosuppressive drug anti-T-lymphocyte globulin; they were also given lower doses of a cytostatic called busulfan, levels of which were regularly measured in the patients' blood, allowing the dosages to be adjusted to obtain the desired amount. Because the metabolism of and variations in exposure to busulfan are greater in children than in adults, the researchers consider it imperative to monitor blood levels of the drug.

"Our aim is for tailored treatments to one day become standard practice, which will result in higher survival rates and improved quality of life; our results are so good that we have now begun a similar dose study using the same 'old' cytostatics on patients with leukemia," says Professor Hassan, who is also especially appreciative of the years of support given to this research by the Swedish Cancer Society and the Swedish Childhood Cancer Foundation.

He hopes that the technique can eventually be used in all drug-based therapies to optimise efficacy for individual patients.


Story Source:

The above story is based on materials provided by Karolinska Institutet. Note: Materials may be edited for content and length.


Journal Reference:

  1. Tayfun Gόngφr, Pierre Teira, Mary Slatter, Georg Stussi, Polina Stepensky, Despina Moshous, Clementien Vermont, Imran Ahmad, Peter J Shaw, Josι Marcos Telles da Cunha, Paul G Schlegel, Rachel Hough, Anders Fasth, Karim Kentouche, Bernd Gruhn, Juliana F Fernandes, Silvy Lachance, Robbert Bredius, Igor B Resnick, Bernd H Belohradsky, Andrew Gennery, Alain Fischer, H Bobby Gaspar, Urs Schanz, Reinhard Seger, Katharina Rentsch, Paul Veys, Elie Haddad, Michael H Albert and Moustapha Hassan. Reduced-intensity conditioning and HLA-matched haematopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study. The Lancet, October 2013

Cite This Page:

Karolinska Institutet. "Tailored doses of cytostatic improve survival rate after stem cell transplantation." ScienceDaily. ScienceDaily, 23 October 2013. <www.sciencedaily.com/releases/2013/10/131023090722.htm>.
Karolinska Institutet. (2013, October 23). Tailored doses of cytostatic improve survival rate after stem cell transplantation. ScienceDaily. Retrieved September 18, 2014 from www.sciencedaily.com/releases/2013/10/131023090722.htm
Karolinska Institutet. "Tailored doses of cytostatic improve survival rate after stem cell transplantation." ScienceDaily. www.sciencedaily.com/releases/2013/10/131023090722.htm (accessed September 18, 2014).

Share This



More Health & Medicine News

Thursday, September 18, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Artificial Sweetener Could Promote Diabetes

Artificial Sweetener Could Promote Diabetes

Newsy (Sep. 17, 2014) — Doctors once thought artificial sweeteners lacked the health risks of sugar, but a new study says they can impact blood sugar levels the same way. Video provided by Newsy
Powered by NewsLook.com
Ebola Vaccine Trial Gets Underway at Oxford University

Ebola Vaccine Trial Gets Underway at Oxford University

AFP (Sep. 17, 2014) — A healthy British volunteer is to become the first person to receive a new vaccine for the Ebola virus after US President Barack Obama called for action against the epidemic and warned it was "spiralling out of control." Duration: 01:02 Video provided by AFP
Powered by NewsLook.com
Obesity Rates Steady Even As Americans' Waistlines Expand

Obesity Rates Steady Even As Americans' Waistlines Expand

Newsy (Sep. 17, 2014) — Researchers are puzzled as to why obesity rates remain relatively stable as average waistlines continue to expand. Video provided by Newsy
Powered by NewsLook.com
President To Send 3,000 Military Personnel To Fight Ebola

President To Send 3,000 Military Personnel To Fight Ebola

Newsy (Sep. 16, 2014) — President Obama is expected to send 3,000 troops to West Africa as part of the effort to contain Ebola's spread. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
 
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:  

Breaking News:
from the past week

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:  

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile iPhone Android Web
Follow Facebook Twitter Google+
Subscribe RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins