Clinical Trial

[vikasgoyal]

My dad is getting transfused with red cells every 6weeks which is really hampering his quality of life. Unfortunately we have not found any good treatment available that can help his hgb counts improve. Rest of his counts have remained stable and he his able to lead his normal life untill it gets to a point where he needs to get transfused. He lives in India and we do not have many clinical trials available there. What is confusing to me right now are couple of questions:

1. Should he try clinical trials at this stage? If yes, being an indian citizen is there a possibility for him to enroll in any of the clinical trials in US ?
2. Is there any low toxic treatment available to improve his hgb?

[triumphe64]

Does he possibly have pure red cell aplasia? It is treated quite differently than MDS.

It only affects the red cells and is very rare. If I understand what you are saying, the red cells are the only problem.

[Birgitta-A]

Hi vikasgoyal,
As far as I understand your father has low risk MDS and only needs txs every 6th week. Most patients with with these symptoms get supportive therapy like txs, EPO and iron chelation drugs like Desferal or Exjade. Then your father is getting Danazol too.

Has his doctor not discussed iron chelation? Some patients get increased HGB when the ferritin level decreases eventually due to less oxidative stress in the bone marrow? The ferritin level should not exceed 1000.

He could try Thalidomide (about 30% respond) or Revlimid (about 60% of patients without chromosome aberrations respond). None of these drugs are low toxic but I have tried both in very low doses with positive effects with low neutrophils (treated with Neupogen) as the only adverse effect.
Kind regards
Birgitta-A
74 yo, dx MDS Interm-1 2006. Supportive therapy until 2010 when I started to take Thalidomide + Prednisone. Transfusion independent during a little more than 2 years. Started Revlimid + Prednisone July 2013.

[vikasgoyal]

He is taking wheatgrass which helped him reduce his ferritin from 2500 to 750 in couple of months. I hope this trend continues. He got his BMB done twice and they confirmed MDS RCMD everytime. One of the doctors told us that for non del 5q cases revlimid might work for only about 20% of the cases. Is that true?

Secondly, is clinical trial a good option if he can be accepted in US?

[triumphe64]

Originally Posted by vikasgoyal

He is taking wheatgrass which helped him reduce his ferritin from 2500 to 750 in couple of months. I hope this trend continues. He got his BMB done twice and they confirmed MDS RCMD everytime. One of the doctors told us that for non del 5q cases revlimid might work for only about 20% of the cases. Is that true?

Secondly, is clinical trial a good option if he can be accepted in US?

Clinical trials are all listed at:

www.clinicaltrials.gov

See is any fit.

[Birgitta-A]

Hi vikasgoyal,
Very positive result with wheatgrass!

I have results for many studies of Revlimid in non 5q MDS patients - they all show very different results. Here is one with 57% response rate for patients with normal chromosomes: http://www.nejm.org/doi/full/10.1056/NEJMoa041668
Kind regards
Birgitta-A
12p- and X- chromosome aberrations

[vikasgoyal]

Hi Birgitta-A,

Thanks for the response. Does revlimid has more significance in improving plt or hgb?

Thanks,
Vikas

[Birgitta-A]

Hi vikasgoyal,
Revlimid is used to increase HGB. Myelosuppression (suppression of the bone marrow) is a common adverse effect for patients with the 5q chromosome aberration and low platelets initially is a sign of positive response for these patients.

In non 5q patients myelosupression often isn´t so severe.
http://bloodjournal.hematologylibrar.../1/86.full.pdf

Revlimid should be combined with Prednisone (30 mg/day) in tapering dose for better response.
Kind regards
Birgitta-A

[vikasgoyal]

Thanks..Great info